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Solid Biosciences to Present at Upcoming Scientific Meetings
Globenewswire· 2025-10-01 12:00
CHARLESTOWN, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from its neuromuscular and cardiac programs at the World Muscle Society (WMS) 2025 Annual International Congress, October 7-11, 2025, in Vienna, Austria, and at the European Society of Gene & Cell Therapy (ESGCT) 2025 Annual Congress, October 7-12, 2025, in Seville, Spain. “Our presentations at WM ...
Solid Biosciences to Present at 2025 Neuromuscular Study Group Annual Scientific Meeting
Globenewswire· 2025-09-25 12:00
CHARLESTOWN, Mass., Sept. 25, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation investigational gene therapy intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 26th Annual Scientific Meeting of Neuromuscular Study Group (NMSG), September 26-28, 2025, in Stresa, Italy. Pr ...
Solid Biosciences Announces Licensing Agreement with Kinea Bio for the Use of Proprietary Next-Generation Capsid AAV-SLB101
Globenewswire· 2025-09-23 12:00
- Non-exclusive license for Solid’s proprietary, next generation capsid, AAV-SLB101, to accelerate development of Kinea Bio’s gene therapy for dysferlin-related limb-girdle muscular dystrophy - - Solid to receive an upfront payment and is eligible for certain development and sales milestones and tiered royalties on net sales -- Solid continues to expand collaborative efforts for AAV-SLB101 with more than 25 agreements and licenses executed to date - CHARLESTOWN, Mass., Sept. 23, 2025 (GLOBE NEWSWIRE) -- Sol ...
Solid Biosciences (NasdaqGS:SLDB) 2025 Conference Transcript
2025-09-09 20:12
Summary of Solid Biosciences Conference Call Company Overview - **Company**: Solid Biosciences (NasdaqGS:SLDB) - **Focus**: Precision genetic medicine, primarily in gene therapy targeting neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (DMD), Friedreich's ataxia, and catecholaminergic polymorphic ventricular tachycardia (CPVT) [2][4] Key Programs - **SGT-003**: Targeting Duchenne muscular dystrophy, currently in clinical trials with 15 patients dosed, showing positive early results [4][8] - **Friedreich's Ataxia Program**: Utilizing dual-route administration to address both CNS and cardiac manifestations [30][31] - **SGT-401**: Focused on CPVT, aiming to regulate calcium signaling to prevent arrhythmias [39][41] - **SGT-601**: Targeting TNNT2 dilated cardiomyopathy, currently in GLP toxicology [2][42] Clinical Data and Efficacy - **DMD Clinical Data**: - 15 patients dosed with SGT-003, showing no serious adverse events (SAEs) and positive biomarkers [12][11] - Achieved a target of over three vector genomes per nucleus, indicating strong expression levels [9][10] - Positive changes in cardiac biomarkers, including ejection fraction and troponin levels [11][12] - **Safety Profile**: - Clean safety profile with only one grade 1 liver enzyme spike reported, managed effectively [15][16] - Focus on maintaining low doses and high full-to-empty capsid ratios to minimize liver-related risks [14][15] Regulatory Strategy - **FDA Engagement**: Plans to meet with the FDA in Q4 to discuss pivotal trial design and potential for accelerated approval [20][21] - **Trial Design**: Proposed multi-country, multi-site, double-blind trial with endpoints including time to rise and cardiac output metrics [25][26] Financial Position - **Current Cash Position**: Approximately $268 million as of Q3, expected to fund operations into the first half of 2027 [43] - **Partnerships**: Open to partnerships but confident in the company's ability to advance independently [43] Industry Context and Innovation - **Gene Therapy Landscape**: Emphasis on advancing gene therapy through innovative capsids, promoters, and manufacturing processes to improve delivery and efficacy [45][46] - **Collaborations**: Currently engaged in 26 collaborations aimed at enhancing gene therapy tools, with a goal of reaching 100 [46][47] Additional Insights - **Market Positioning**: Solid Biosciences aims to elevate the entire gene therapy market by providing advanced tools and technologies to other companies, thereby fostering broader investment in the sector [46][47] This summary encapsulates the key points discussed during the conference call, highlighting Solid Biosciences' strategic focus, clinical advancements, regulatory plans, financial health, and commitment to innovation in gene therapy.
Solid Biosciences (NasdaqGS:SLDB) FY Conference Transcript
2025-09-08 14:02
Summary of Solid Biosciences FY Conference Call Company Overview - **Company**: Solid Biosciences (NasdaqGS:SLDB) - **Focus**: Clinical-stage biopharmaceutical company developing AAV-based gene therapies for rare neuromuscular and cardiac genetic diseases [2][4] Pipeline Overview - **Key Products**: - **SGT-003**: Next-generation gene therapy for Duchenne muscular dystrophy (DMD) - **Friedreich's Ataxia**: First-ever dual route of administration - **CPVT**: Treatment for catecholaminergic polymorphic ventricular tachycardia - **TNNT2**: Targeting dilated cardiomyopathy affecting 20,000 to 27,000 patients [4][5] Clinical Results and Safety Profile - **SGT-003**: - Encouraging data from the ongoing INSPIRE study - 15 boys dosed safely with no serious adverse events (SAEs) or hospitalizations reported [5][8][12] - Focus on a holistic approach to data analysis, considering multiple biomarkers beyond just protein levels [6][9] - Novel capsid SLB-101 shows liver detargeting and improved biodistribution compared to AAV9, with expression 20 times greater in cardiomyocytes [9][10][11] Regulatory Strategy - **FDA Interaction**: Plans to present a robust data package to the FDA in the upcoming quarter [13][14] - **International Trials**: Initiating a double-blind, placebo-controlled trial outside the U.S. to ensure global access and meet regulatory requirements [18][19] Market Potential and Treatment Landscape - **Duchenne Muscular Dystrophy**: SGT-003 aims to provide families with treatment options, addressing the critical need for effective therapies [22][23] - **Friedreich's Ataxia**: Unique dual route administration targeting both CNS and cardiac manifestations, with potential to dominate the market if proven safe and effective [28][30] Future Plans and Milestones - **Upcoming Trials**: Plans to dose patients in Friedreich's Ataxia and CPVT by Q4 2025 [31][37] - **Cash Position**: As of August, the company has approximately $268 million in cash, sufficient to support ongoing trials and operations [37] Additional Insights - **Cautious Expansion**: The company will not expand into non-ambulatory patients until safety is well established in the current cohort [26][27] - **Trial Design Considerations**: Emphasis on specific endpoints like Stride Velocity for efficacy assessment in upcoming trials [20][21] This summary encapsulates the key points discussed during the conference call, highlighting Solid Biosciences' strategic focus, clinical advancements, regulatory plans, and market potential.
Solid Biosciences (SLDB) 2025 Conference Transcript
2025-09-04 20:20
Summary of Solid Biosciences (SLDB) 2025 Conference Call Company Overview - Solid Biosciences is a precision genetic medicine company focused on gene therapy, addressing unmet needs in neuromuscular and cardiac diseases. Current programs include Duchenne muscular dystrophy (DMD), Fragile Cachexia, Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), and TNNT2 cardiac program [3][4] Core Points and Arguments Safety Considerations - Safety is a primary focus in all programs, with efforts to mitigate risks through improved delivery methods, including capsids and promoters [4][5] - 15 boys have been dosed in the Duchenne program with no serious adverse events (SAEs) or hospitalizations reported [17][18] - The company emphasizes the importance of purity in manufacturing over yield, as higher purity can lead to better safety and efficacy outcomes [14][15] Efficacy and Clinical Data - The company believes that the percentage of positive fibers in muscle tissue is critical for assessing clinical benefit, aiming for a target of 40% positive fibers [20][21] - The approach includes a holistic view of various biomarkers to assess muscle integrity and overall impact [21][23] - The company plans to meet with the FDA to discuss the path for accelerated approval based on safety and biochemical changes observed in trials [25][27] CPVT Program - CPVT is a fatal arrhythmia condition often misdiagnosed, with an estimated 20,000 patients potentially affected by specific mutations [29][30] - Solid Biosciences is developing a treatment that targets excess calcium in heart muscles, which is a novel approach compared to existing therapies [32][33] Financial Position - The company has approximately $268 million in cash, expected to last until mid-2027, and is not seeking to raise funds in the near term [43] Other Important Content - The company is focused on licensing its capsids to small companies and academic labs, aiming for a significant market presence in cardiac and neuromuscular programs over the next three to five years [39][40] - The CEO expressed confidence in the company's unique approach and the potential for significant advancements in gene therapy [43][44]
Solid Biosciences Inc. (SLDB) Presents At Citi's Biopharma Back To School Conference Transcript
Seeking Alpha· 2025-09-02 20:28
Question-and-Answer SessionSo let's just start with just an overview question, if we could, just introduce the company, the focus of the company, what are some of the key programs, and then we can get into detail on each of them.Alexander CumboPresident, CEO & Director Yes. Thank you, [indiscernible] for the invitation. Solid Biosciences is a precision genetic medicine company. We primarily focus right now on gene therapy. Three main drugs that are going to be either -- that are in the clinic, IND is open a ...
Solid Biosciences (SLDB) Conference Transcript
2025-09-02 18:47
Summary of Solid Biosciences Conference Call Company Overview - **Company**: Solid Biosciences (SLDB) - **Focus**: Precision genetic medicine, primarily gene therapy - **Key Programs**: - Duchenne muscular dystrophy (DMD) - Friedreich's ataxia (FA) - Catecholaminergic polymorphic ventricular tachycardia (CPVT) - Upcoming program for dilated cardiomyopathy (TNNT2) in 2026 - **Employee Count**: Approximately 110 employees based in Boston [2][2] Core Points and Arguments Duchenne Muscular Dystrophy (DMD) Program - **Unique Properties**: - Unique capsid and construct with R16, R17 domain for enhanced blood flow and reduced inflammation [4][4] - Modified AAV9 capsid with RGD peptides targeting skeletal and cardiac muscle, showing 20-fold greater cardiomyocyte targeting compared to AAV9 [5][5] - **Dosing and Safety**: - 15 boys aged 5 to 10 have been dosed, with positive safety outcomes including transient nausea and vomiting [8][8] - Fast tapering of steroids post-dosing, with 93% able to taper from day 30 to day 60 [9][9] - **Expression Data**: - High levels of vector genome copies observed, with a focus on positive fiber counts for assessing efficacy [10][10] - Emphasis on muscle integrity and biomarkers like ALT, AST, and troponin to monitor cardiac function [12][13] Upcoming FDA Meeting - **Goals**: Present data and seek a path for accelerated approval, aiming for a registrational study by year-end [21][22] - **Proposed Parameters**: 30-40 patients for safety database, 10% mean expression, and directional clinical benefit compared to natural history [23][24] Friedreich's Ataxia (FA) Program - **Target Population**: Initially targeting patients aged 18 and above, with plans to include younger patients [54][54] - **Administration Method**: Dual-route administration (IV and direct injection into the cerebellum) [55][55] - **Timeline**: First patient dosing expected in Q4 2025, with results anticipated in the first half of 2026 [61][61] Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) Program - **Disease Overview**: Characterized by calcium overload due to mutations in the ryanodine channel, leading to arrhythmias [68][68] - **Market Need**: Approximately 20,000 patients in the U.S. with no current drug treatment available [71][71] - **Study Start**: Clinical trials for CPVT expected to begin in Q4 2025 [75][75] Dilated Cardiomyopathy (TNNT2) Program - **Status**: Preclinical data is promising, with no current market drugs or trials available for this condition [76][76] Other Important Insights - **Community Feedback**: Physicians and families show strong belief in gene therapy, with many families eager to participate in trials [48][50] - **Combination Therapy Outlook**: Long-term vision includes potential combination therapies with existing treatments like Skyclarys [52][52] - **Regulatory Challenges**: Emphasis on the importance of a clean safety profile for successful reimbursement and market access [46][46] This summary encapsulates the key points discussed during the Solid Biosciences conference call, highlighting the company's focus on innovative gene therapies and the strategic plans for upcoming clinical trials and regulatory engagements.
Solid Biosciences Reports Second Quarter 2025 Financial Results and Provides Business Updates
Globenewswire· 2025-08-12 20:07
Core Insights - Solid Biosciences is advancing its pipeline with three clinical-stage programs targeting neuromuscular and cardiac diseases, including SGT-003 for Duchenne Muscular Dystrophy, SGT-212 for Friedreich's Ataxia, and SGT-501 for Catecholaminergic Polymorphic Ventricular Tachycardia [2][4][9] Clinical Trials and Pipeline - The Phase 1/2 INSPIRE DUCHENNE trial has dosed 15 participants, with ongoing recruitment and no serious adverse events reported [1][3][5] - SGT-212 and SGT-501 are expected to initiate Phase 1b trials in Q4 2025, with SGT-212 being the first gene therapy for Friedreich's Ataxia utilizing a dual route of administration [1][4][6][9] - A randomized, double-blind, placebo-controlled trial for SGT-003 is planned outside the U.S. in Q4 2025 to support global regulatory authorizations [5] Financial Position - As of June 30, 2025, the company reported $268.1 million in cash and equivalents, providing a cash runway into H1 2027 [1][8] - Research and Development expenses for Q2 2025 were $32.4 million, up from $19.5 million in Q2 2024, primarily due to increased costs associated with SGT-003 [8][13] - The net loss for Q2 2025 was $39.5 million, compared to $25.1 million in Q2 2024, reflecting increased operational expenses [13][15] Strategic Partnerships and Technology - Solid has executed over 25 agreements for the use of its proprietary AAV-SLB101 capsid with academic labs and institutions [1][9] - The company is developing multiple next-generation capsid and promoter libraries, with the first cardiac capsid library selection expected in Q4 2025 [9] Market Position and Mission - Solid Biosciences is focused on improving the lives of patients with rare diseases, leveraging its expertise in genetic medicine to innovate therapies for conditions like Duchenne and Friedreich's Ataxia [2][11]
Solid Biosciences(SLDB) - 2025 Q2 - Quarterly Report
2025-08-12 20:03
[Cautionary Note Regarding Forward-Looking Statements and Industry Data](index=4&type=section&id=CAUTIONARY%20NOTE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS%20AND%20INDUSTRY%20DATA) This section provides important disclaimers about forward-looking statements and industry data, highlighting inherent risks and uncertainties [Forward-Looking Statements](index=4&type=section&id=Forward-Looking%20Statements) This section highlights that the report contains forward-looking statements, identifiable by specific terminology, which involve inherent risks and uncertainties - Forward-looking statements are based on operating budgets and forecasts, but actual results may differ materially due to unpredictable factors[9](index=9&type=chunk)[10](index=10&type=chunk) - Key areas of forward-looking statements include timing and results of preclinical/clinical trials (SGT-003, SGT-212, SGT-401, SGT-501, SGT-601), regulatory approvals, market opportunities, manufacturing, commercialization plans, pricing/reimbursement, platform technologies, capital resources, expenses, intellectual property, competitive position, and impact of laws/regulations[11](index=11&type=chunk) - The report's industry and market data are derived from third-party publications and internal estimates, which involve assumptions and limitations, and readers are cautioned against undue reliance[13](index=13&type=chunk) [PART I. FINANCIAL INFORMATION](index=6&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This part presents the company's unaudited condensed consolidated financial statements and management's discussion and analysis of financial condition [Item 1. Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents unaudited condensed consolidated financial statements, including balance sheets, statements of operations, and cash flows [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Presents the company's financial position, detailing assets, liabilities, and equity at specific reporting dates | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | :--------- | | Cash and cash equivalents | $138,933 | $80,235 | $58,698 | 73.16% | | Available-for-sale securities | $129,177 | $68,685 | $60,492 | 88.07% | | Total current assets | $278,640 | $157,302 | $121,338 | 77.14% | | Total assets | $308,292 | $188,662 | $119,630 | 63.41% | | Total liabilities | $50,001 | $51,416 | $(1,415) | -2.75% | | Total stockholders' equity | $258,291 | $137,246 | $121,045 | 88.20% | [Condensed Consolidated Statements of Operations](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Details the company's revenues, expenses, and net loss over specific reporting periods | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :-------------------- | :--------- | | Research and development | $32,415 | $19,461 | $12,954 | 66.6% | | General and administrative | $9,278 | $8,327 | $951 | 11.4% | | Total operating expenses | $41,693 | $27,788 | $13,905 | 50.0% | | Net loss | $(39,480) | $(25,072) | $(14,408) | 57.5% | | Net loss per share, basic and diluted | $(0.42) | $(0.61) | $0.19 | -31.1% | | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :-------------------- | :--------- | | Research and development | $63,329 | $38,334 | $24,995 | 65.2% | | General and administrative | $18,416 | $16,316 | $2,100 | 12.9% | | Total operating expenses | $81,745 | $54,650 | $27,095 | 49.6% | | Net loss | $(78,762) | $(49,375) | $(29,387) | 59.5% | | Net loss per share, basic and diluted | $(0.98) | $(1.25) | $0.27 | -21.6% | [Condensed Consolidated Statements of Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) Reports the company's net loss and other comprehensive income/loss components for the periods presented | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :-------------------- | :--------- | | Net loss | $(39,480) | $(25,072) | $(14,408) | 57.5% | | Unrealized loss on available-for-sale securities | $(40) | $(11) | $(29) | 263.6% | | Comprehensive loss | $(39,520) | $(25,083) | $(14,437) | 57.6% | | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :-------------------- | :--------- | | Net loss | $(78,762) | $(49,375) | $(29,387) | 59.5% | | Unrealized loss on available-for-sale securities | $(85) | $(30) | $(55) | 183.3% | | Comprehensive loss | $(78,847) | $(49,405) | $(29,442) | 59.6% | [Condensed Consolidated Statements of Changes in Stockholders' Equity](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Stockholders'%20Equity) Outlines changes in the company's equity, including stock issuances and net loss, over the reporting period | Metric | January 1, 2025 (in thousands) | June 30, 2025 (in thousands) | Change (in thousands) | | :------------------------------------------------ | :----------------------------- | :--------------------------- | :-------------------- | | Total Stockholders' Equity | $137,246 | $258,291 | $121,045 | | Issuance of common stock in public offering, net | - | $135,367 | $135,367 | | Issuance of pre-funded warrants in public offering, net | - | $52,590 | $52,590 | | Issuance of common stock in private placement for developmental milestone | - | $5,000 | $5,000 | | Net loss (six months) | $(78,762) | - | $(78,762) | - The company issued **35,739,810 shares of common stock** and pre-funded warrants for **13,888,340 shares** in a February 2025 public offering, generating approximately **$188.0 million in net proceeds**[24](index=24&type=chunk)[61](index=61&type=chunk) - **975,496 shares of common stock** were issued as payment for a **$5.0 million developmental milestone** to FA212 on February 28, 2025[24](index=24&type=chunk)[50](index=50&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Summarizes cash inflows and outflows from operating, investing, and financing activities | Cash Flow Activity | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | Change (in thousands) | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :-------------------- | | Net cash used in operating activities | $(69,262) | $(46,659) | $(22,603) | | Net cash used in investing activities | $(60,195) | $(43,666) | $(16,529) | | Net cash provided by financing activities | $188,127 | $112,241 | $75,886 | | Net increase in cash, cash equivalents, and restricted cash | $58,670 | $21,916 | $36,754 | | Cash, cash equivalents, and restricted cash at end of period | $140,857 | $97,764 | $43,093 | - Financing activities in H1 2025 were significantly boosted by **$189.6 million from public offerings** of common stock and pre-funded warrants, net of underwriting discounts and commissions[29](index=29&type=chunk)[141](index=141&type=chunk) - Non-cash investing activity included **$5.0 million in common stock** issued as payment for developmental milestone consideration in H1 2025[29](index=29&type=chunk) [Notes to the Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20the%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations of accounting policies, significant transactions, and financial statement line items [1. Nature of the Business and Basis of Presentation](index=13&type=section&id=1.%20Nature%20of%20the%20Business%20and%20Basis%20of%20Presentation) Describes the company's core business, gene therapy candidates, and the basis for financial statement preparation - The company is focused on advancing gene therapy candidates for Duchenne muscular dystrophy (SGT-003), Friedreich's ataxia (SGT-212), catecholaminergic polymorphic ventricular tachycardia (SGT-501), and TNNT2-mediated dilated cardiomyopathy (SGT-601), among others[34](index=34&type=chunk) - As of June 30, 2025, the company had **$268.1 million in cash, cash equivalents, and available-for-sale securities** (excluding restricted cash), which is expected to fund operations and capital expenditures for at least twelve months from the issuance date of the statements[37](index=37&type=chunk) - The company expects to continue generating operating losses and will need to finance future cash needs through equity offerings, debt financings, collaborations, or licensing arrangements[39](index=39&type=chunk) [2. FA212 Asset Acquisition](index=14&type=section&id=2.%20FA212%20Asset%20Acquisition) Details the acquisition of intellectual property for SGT-212, including upfront and contingent payments - The company acquired intellectual property for SGT-212 in September 2024, including patents and manufacturing contracts, for an **upfront payment of $1.0 million**[46](index=46&type=chunk)[47](index=47&type=chunk) - Contingent development milestone payments to FA212 of up to **$34.0 million** and sales milestone payments of up to **$21.0 million**, plus tiered royalties, were agreed upon[47](index=47&type=chunk) - The IND for SGT-212 was cleared by the FDA in December 2024, leading to a **$5.0 million development milestone payment** to FA212, satisfied by issuing **975,496 shares of common stock** on February 28, 2025[50](index=50&type=chunk) [3. Fair Value Measurements](index=15&type=section&id=3.%20Fair%20Value%20Measurements) Explains the valuation methodologies and inputs used for financial instruments measured at fair value | Financial Instrument | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Fair Value Level | | :-------------------------------- | :----------------------------- | :------------------------------- | :--------------- | | Cash equivalents | $130,331 | $35,522 | Level 2 | | Available-for-sale securities | $129,177 | $68,685 | Level 2 | | Derivative liabilities | $5,700 | $3,150 | Level 3 | - Derivative liabilities are valued using a Monte Carlo simulation, with key unobservable inputs including probability of achieving development milestones (**28.0%-70.0%**), volatility (**93.0%** at June 30, 2025), and discount rate (**3.7%-4.3%**)[53](index=53&type=chunk)[54](index=54&type=chunk) - The change in fair value of derivative liabilities resulted in losses of **$0.9 million** for the three months and **$2.6 million** for the six months ended June 30, 2025[54](index=54&type=chunk) [4. Available-for-Sale Securities](index=17&type=section&id=4.%20Available-for-Sale%20Securities) Provides details on the company's available-for-sale investment portfolio, including fair values and maturities | Security Type | June 30, 2025 Fair Value (in thousands) | December 31, 2024 Fair Value (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :-------------------- | :--------- | | Treasury bills | $42,287 | $27,895 | $14,392 | 51.6% | | Government bonds | $86,890 | $40,790 | $46,100 | 113.0% | | Total available-for-sale securities | $129,177 | $68,685 | $60,492 | 88.1% | - The weighted average contractual maturity of available-for-sale securities was approximately **0.6 years** as of June 30, 2025[57](index=57&type=chunk) [5. Prepaid Expenses and Other Current Assets](index=19&type=section&id=5.%20Prepaid%20Expenses%20and%20Other%20Current%20Assets) Details the composition and changes in prepaid expenses and other current assets | Category | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | :--------- | | Prepaid research and development expenses | $7,235 | $6,731 | $504 | 7.5% | | Prepaid other and other current assets | $3,295 | $1,651 | $1,644 | 99.6% | | Total | $10,530 | $8,382 | $2,148 | 25.6% | [6. Accrued Expenses and Other Current Liabilities](index=19&type=section&id=6.%20Accrued%20Expenses%20and%20Other%20Current%20Liabilities) Outlines the various accrued expenses and other current liabilities of the company | Category | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | :--------- | | Accrued research and development expenses | $9,944 | $7,268 | $2,676 | 36.8% | | Accrued compensation | $4,618 | $6,221 | $(1,603) | -25.8% | | Accrued milestone payment | $0 | $5,000 | $(5,000) | -100.0% | | Accrued other and other current liabilities | $1,984 | $1,363 | $621 | 45.6% | | Total | $16,546 | $19,852 | $(3,306) | -16.7% | [7. Stockholders' Equity](index=19&type=section&id=7.%20Stockholders'%20Equity) Describes changes in common stock, pre-funded warrants, and overall stockholders' equity - Common stock authorized shares increased from **120,000,000 to 240,000,000** at June 30, 2025[18](index=18&type=chunk) - In February 2025, the company issued **35,739,810 shares of common stock** and pre-funded warrants to purchase **13,888,340 shares**, generating approximately **$188.0 million in net proceeds**[61](index=61&type=chunk) - Pre-funded warrants are classified as equity and none were exercised during the six months ended June 30, 2025[62](index=62&type=chunk) [8. Equity-Based Compensation](index=19&type=section&id=8.%20Equity-Based%20Compensation) Details the company's equity incentive plans and associated compensation expenses [Equity-Based Compensation Expense](index=19&type=section&id=Equity-Based%20Compensation%20Expense) Summarizes the equity-based compensation expenses recognized across different categories | Category | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :-------------------- | :--------- | | Research and development | $1,116 | $832 | $284 | 34.1% | | General and administrative | $2,029 | $1,260 | $769 | 61.0% | | Total | $3,145 | $2,092 | $1,053 | 50.3% | | Category | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :-------------------- | :--------- | | Research and development | $2,218 | $1,380 | $838 | 60.7% | | General and administrative | $4,268 | $2,323 | $1,945 | 83.7% | | Total | $6,486 | $3,703 | $2,783 | 75.2% | [Equity Incentive Plans](index=20&type=section&id=Equity%20Incentive%20Plans) Describes the various equity incentive plans and shares available for future issuance - The company's equity incentive plans include the 2018 Omnibus Incentive Plan, Amended and Restated 2020 Equity Incentive Plan, Amended and Restated 2021 Employee Stock Purchase Plan, and 2024 Inducement Stock Incentive Plan[64](index=64&type=chunk) - On June 12, 2025, stockholders approved an amendment to the 2020 Plan, increasing the number of shares reserved for issuance by **9,000,000**[65](index=65&type=chunk) - As of June 30, 2025, **9,173,225 shares** remained available for future issuance under the 2020 Plan, and **386,111 shares** under the 2024 Plan[65](index=65&type=chunk)[66](index=66&type=chunk) [Stock Options](index=20&type=section&id=Stock%20Options) Provides details on outstanding stock options, their exercise prices, and remaining contractual lives | Metric | January 1, 2025 | June 30, 2025 | | :-------------------------------- | :-------------- | :------------ | | Options Outstanding | 2,927,937 | 3,324,305 | | Weighted Average Exercise Price | $18.22 | $13.93 | | Weighted Average Remaining Contractual Life (years) | 8.01 | 8.14 | - Equity-based compensation expense for stock options was **$2.6 million** for the six months ended June 30, 2025, up from **$2.0 million** in the prior year[68](index=68&type=chunk) - As of June 30, 2025, **$8.3 million** of unrecognized equity-based compensation expense related to unvested stock options is expected to be recognized over a weighted average period of **1.7 years**[68](index=68&type=chunk) [Restricted Stock Units](index=21&type=section&id=Restricted%20Stock%20Units) Outlines the activity and unrecognized expense related to restricted stock units | Metric | January 1, 2025 | June 30, 2025 | | :-------------------------------- | :-------------- | :------------ | | Nonvested RSUs | 1,480,272 | 3,102,124 | | Granted RSUs | - | 2,188,256 | | Vested RSUs | - | (280,159) | - Equity-based compensation expense for RSUs was **$2.1 million** for the six months ended June 30, 2025, up from **$1.4 million** in the prior year[69](index=69&type=chunk) - As of June 30, 2025, **$12.6 million** of unrecognized equity-based compensation expense related to nonvested RSUs is expected to be recognized over a weighted average period of **3.2 years**[69](index=69&type=chunk) [Performance Stock Units](index=21&type=section&id=Performance%20Stock%20Units) Details the nonvested performance stock units and associated compensation expense | Metric | January 1, 2025 | June 30, 2025 | | :-------------------------------- | :-------------- | :------------ | | Nonvested PSUs | 2,165,325 | 2,074,188 | | Forfeitures | - | (91,137) | - Equity-based compensation expense for PSUs was **$1.8 million** for the six months ended June 30, 2025, significantly up from **$0.1 million** in the prior year[72](index=72&type=chunk) - As of June 30, 2025, **$3.8 million** of unrecognized equity-based compensation expense related to nonvested PSUs is expected to be recognized over a weighted average period of **1.4 years**, assuming all Performance Milestones are achieved[73](index=73&type=chunk) [9. Commitments and Contingencies](index=21&type=section&id=9.%20Commitments%20and%20Contingencies) Discusses the company's contractual obligations, indemnification agreements, and legal proceedings [Letter of Credit](index=21&type=section&id=Letter%20of%20Credit) Reports the outstanding balance of the company's letter of credit - Outstanding letter of credit was **$1.9 million** at June 30, 2025, down from **$2.0 million** at December 31, 2024[74](index=74&type=chunk) [Indemnification Agreements](index=22&type=section&id=Indemnification%20Agreements) Describes the company's indemnification obligations to officers and directors - The company provides indemnification to various parties, including executive officers and board members, for certain liabilities, with maximum potential payments often unlimited[75](index=75&type=chunk) - No material costs have been incurred from indemnification arrangements as of June 30, 2025, and December 31, 2024[76](index=76&type=chunk) [Legal Proceedings](index=22&type=section&id=Legal%20Proceedings) States the company's awareness of any material legal proceedings or claims - The company is not aware of any material legal proceedings or claims as of June 30, 2025[77](index=77&type=chunk) [10. Net Loss Per Share](index=22&type=section&id=10.%20Net%20Loss%20Per%20Share) Calculates basic and diluted net loss per share, including weighted average shares outstanding | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Net loss per share, basic and diluted | $(0.42) | $(0.61) | | Weighted average shares of common stock outstanding | 77,539,468 | 38,221,883 | | Weighted average shares of pre-funded warrants | 16,600,818 | 2,712,478 | | Total weighted average shares | 94,140,286 | 40,934,361 | | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | | Net loss per share, basic and diluted | $(0.98) | $(1.25) | | Weighted average shares of common stock outstanding | 67,476,597 | 36,981,426 | | Weighted average shares of pre-funded warrants | 12,840,991 | 2,563,441 | | Total weighted average shares | 80,317,588 | 39,544,867 | - Pre-funded warrants are considered outstanding for basic and diluted net loss per share calculation due to exercisability for nominal consideration[78](index=78&type=chunk) [11. Segment Reporting](index=23&type=section&id=11.%20Segment%20Reporting) Identifies the company's operating segment and how performance is assessed by management - The company has one reportable and one operating segment, focused on developing treatments for neuromuscular and cardiac diseases[81](index=81&type=chunk) - The chief executive officer is the chief operating decision maker, assessing performance and allocating resources based on net loss[82](index=82&type=chunk) | Segment Expense Category | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :---------------------------------- | :---------------------------------- | | SGT-003 | $12,219 | $2,434 | $21,385 | $4,340 | | SGT-501 | $2,223 | $3,422 | $6,689 | $7,229 | | External R&D other | $3,116 | $1,845 | $5,886 | $4,573 | | Internal R&D expense | $6,765 | $4,921 | $13,608 | $10,298 | | External G&A expense | $5,279 | $5,449 | $10,156 | $10,883 | | Internal G&A expense | $3,360 | $3,037 | $6,823 | $6,003 | | Other segment items | $8,731 | $6,680 | $17,198 | $11,324 | | Other income, net | $(2,213) | $(2,716) | $(2,983) | $(5,275) | | Condensed consolidated net loss | $(39,480) | $(25,072) | $(78,762) | $(49,375) | [12. Subsequent Events](index=23&type=section&id=12.%20Subsequent%20Events) Discusses significant events occurring after the balance sheet date, such as new tax laws - The OBBBA, enacted July 4, 2025, includes significant changes to federal tax law and other regulatory provisions[85](index=85&type=chunk) - The company is evaluating the impact of the OBBBA on its forecasted annual effective tax rate in subsequent periods[85](index=85&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management's perspective on financial condition, operational results, pipeline progress, and future funding needs [Overview](index=24&type=section&id=Overview) Introduces the company's gene therapy pipeline, corporate vision, and key designations for candidates - The company's pipeline includes SGT-003 for Duchenne, SGT-212 for Friedreich's ataxia, SGT-501 for CPVT, and SGT-601 for TNNT2-mediated dilated cardiomyopathy[90](index=90&type=chunk) - SGT-003 has Fast Track, Orphan Drug, and Rare Pediatric Disease designations; SGT-212 has Fast Track; and SGT-501 has Fast Track, Orphan Drug, and Rare Pediatric Disease designations[92](index=92&type=chunk) - The corporate vision is to build an innovation platform for high-value genetic medicines for neuromuscular and cardiac diseases, integrating internal capabilities and collaborations[91](index=91&type=chunk) [SGT-003](index=26&type=section&id=SGT-003) Details the clinical trial progress, initial data, and regulatory plans for the Duchenne muscular dystrophy candidate - Participant dosing in the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003 began in Q2 2024, with **15 participants dosed** as of August 12, 2025[95](index=95&type=chunk)[97](index=97&type=chunk) - Initial data from the INSPIRE DUCHENNE trial (as of Feb 11, 2025) showed an average microdystrophin expression of **110% by western blot** and improvements in muscle integrity biomarkers in the first three participants[96](index=96&type=chunk) - The company anticipates a meeting with the FDA in Q4 2025 to discuss potential regulatory pathways for SGT-003 and plans to initiate a separate randomized, double-blind, placebo-controlled trial outside the U.S. in Q4 2025[98](index=98&type=chunk)[99](index=99&type=chunk) [SGT-212](index=26&type=section&id=SGT-212) Outlines the regulatory clearance and anticipated clinical trial initiation for the Friedreich's ataxia candidate - The FDA cleared the IND for SGT-212 for Friedreich's ataxia in January 2025[100](index=100&type=chunk) - An open-label, multi-center Phase 1b clinical trial for SGT-212 is anticipated to begin in Q4 2025, enrolling adult participants with FA to evaluate safety and tolerability[100](index=100&type=chunk) - SGT-212 has been granted Fast Track designation by the FDA for the treatment of FA[100](index=100&type=chunk) [SGT-501](index=26&type=section&id=SGT-501) Reports regulatory approvals and planned clinical trial for the CPVT gene therapy candidate - The FDA cleared the IND and Health Canada approved the CTA for SGT-501 for CPVT in July 2025[101](index=101&type=chunk) - An open-label, multi-center Phase 1b clinical trial for SGT-501 in adult CPVT participants is anticipated to begin in Q4 2025[101](index=101&type=chunk) - SGT-501 has Fast Track, Orphan Drug, and Rare Pediatric Disease designations for CPVT[101](index=101&type=chunk) [Other Cardiac Programs](index=26&type=section&id=Other%20Cardiac%20Programs) Briefly describes preclinical stage cardiac candidates and their efficacy studies - SGT-601 is a preclinical stage product candidate for TNNT2 DCM[102](index=102&type=chunk) - Efficacy studies in mice suggest SGT-601 treatment restored ejection fraction function and stabilized cardiac function over time[102](index=102&type=chunk) [Capsids](index=26&type=section&id=Capsids) Discusses the proprietary AAV-SLB101 capsid and the development of next-generation capsid libraries - AAV-SLB101, Solid's proprietary capsid for SGT-003, has been well tolerated in the INSPIRE DUCHENNE trial (N=15) and in NHP and mouse studies[103](index=103&type=chunk) - The company is building multiple cardiac and neuromuscular next-generation capsid and promoter libraries, with final capsid selection from the first cardiac library anticipated in Q4 2025[105](index=105&type=chunk) - Gene transfer products utilize a viral capsid to deliver functional genes (transgenes) to cells, which then produce therapeutic proteins[104](index=104&type=chunk) [Our Operations](index=27&type=section&id=Our%20Operations) Summarizes net losses, accumulated deficit, and future funding requirements for ongoing operations - The company incurred net losses of **$39.5 million** and **$78.8 million** for the three and six months ended June 30, 2025, respectively, and had an accumulated deficit of **$862.2 million** as of June 30, 2025[106](index=106&type=chunk) - Cash, cash equivalents, and available-for-sale securities of **$268.1 million** (excluding restricted cash) as of June 30, 2025, are expected to fund operating expenses and capital requirements into the first half of 2027[109](index=109&type=chunk) - Significant additional funding will be needed to support continuing operations, likely through equity offerings, debt financings, or collaborations, as the company expects to incur significant expenses and operating losses for the foreseeable future[107](index=107&type=chunk)[108](index=108&type=chunk) [Financial Operations Overview](index=27&type=section&id=Financial%20Operations%20Overview) Provides a high-level summary of the company's revenue and operating expense classifications [Revenue](index=27&type=section&id=Revenue) States the company's current lack of commercial product revenue and future revenue contingencies - The company has not generated any commercial product revenue to date and does not expect to for the foreseeable future[110](index=110&type=chunk) - Future revenue generation is contingent on successful development and marketing approval of its Candidates[110](index=110&type=chunk) [Operating Expenses](index=27&type=section&id=Operating%20Expenses) Classifies operating expenses and highlights personnel costs as a significant component - Operating expenses are classified into research and development and general and administrative[111](index=111&type=chunk) - Personnel costs, including salaries, benefits, bonuses, and equity-based compensation, constitute a significant portion of both expense categories[111](index=111&type=chunk) [Research and Development Expenses](index=28&type=section&id=Research%20and%20Development%20Expenses) Details the composition and changes in research and development expenses by candidate and category | Candidate/Category | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | SGT-003 | $12,318 | $2,435 | $9,883 | 405.9% | | SGT-601 | $2,268 | $186 | $2,082 | 1119.4% | | SGT-212 | $1,145 | $125 | $1,020 | 816.0% | | Personnel related expenses (unallocated) | $8,160 | $5,850 | $2,310 | 39.5% | | Total R&D expenses | $32,415 | $19,461 | $12,954 | 66.6% | | Candidate/Category | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | SGT-003 | $21,616 | $4,341 | $17,275 | 397.9% | | SGT-601 | $4,422 | $411 | $4,011 | 975.9% | | SGT-212 | $1,970 | $173 | $1,797 | 1038.7% | | Personnel related expenses (unallocated) | $16,150 | $11,917 | $4,233 | 35.5% | | Total R&D expenses | $63,329 | $38,334 | $24,995 | 65.2% | - The increase in SGT-003 costs was primarily due to manufacturing, regulatory, and clinical costs, while SGT-601 and SGT-212 increases were related to manufacturing, research, and clinical costs[124](index=124&type=chunk)[128](index=128&type=chunk) [General and Administrative Expenses](index=30&type=section&id=General%20and%20Administrative%20Expenses) Reports the general and administrative expenses and their primary drivers of change | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | General and administrative expenses | $9,278 | $8,327 | $951 | 11.4% | | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | General and administrative expenses | $18,416 | $16,316 | $2,100 | 12.9% | - The increase in G&A expenses was primarily driven by a **$1.2 million increase in personnel-related costs** for the three months and a **$3.0 million increase** for the six months ended June 30, 2025[125](index=125&type=chunk)[129](index=129&type=chunk) [Other Income, Net](index=30&type=section&id=Other%20Income,%20Net) Summarizes other income and expenses, including changes in fair value of derivative liabilities | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | Total other income, net | $2,213 | $2,716 | $(503) | -18.5% | | Change in fair value of derivative liabilities | $(900) | $0 | $(900) | -100.0% | | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | Total other income, net | $2,983 | $5,275 | $(2,292) | -43.5% | | Change in fair value of derivative liabilities | $(2,550) | $0 | $(2,550) | -100.0% | - Interest income increased by **$401 thousand** for the three months and **$50 thousand** for the six months ended June 30, 2025[123](index=123&type=chunk)[127](index=127&type=chunk) [Critical Accounting Policies and Use of Estimates](index=30&type=section&id=Critical%20Accounting%20Policies%20and%20Use%20of%20Estimates) Highlights key accounting policies and the inherent risks of estimates and assumptions - Significant estimates and assumptions are made for research and development expenses, equity-based compensation, and derivative liabilities[122](index=122&type=chunk) - No material changes to critical accounting policies occurred during the six months ended June 30, 2025[120](index=120&type=chunk) - Actual results could differ materially from estimates, judgments, and assumptions used in applying these policies[120](index=120&type=chunk) [Results of Operations](index=31&type=section&id=Results%20of%20Operations) Compares the company's financial performance for the three and six months ended June 30, 2025 and 2024 [Comparison of the Three Months Ended June 30, 2025 and 2024](index=31&type=section&id=Comparison%20of%20the%20Three%20Months%20Ended%20June%2030,%202025%20and%202024) Compares key financial metrics for the three-month periods, including operating expenses and net loss | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | Research and development | $32,415 | $19,461 | $12,954 | 66.6% | | General and administrative | $9,278 | $8,327 | $951 | 11.4% | | Total operating expenses | $41,693 | $27,788 | $13,905 | 50.0% | | Net loss | $(39,480) | $(25,072) | $(14,408) | 57.5% | [Research and Development Expenses (Three Months)](index=31&type=section&id=Research%20and%20Development%20Expenses%20(Three%20Months)) Details the changes in research and development expenses for the three-month periods | Candidate/Category | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | SGT-003 | $12,318 | $2,435 | $9,883 | 405.9% | | SGT-601 | $2,268 | $186 | $2,082 | 1119.4% | | SGT-212 | $1,145 | $125 | $1,020 | 816.0% | | Personnel related expenses (unallocated) | $8,160 | $5,850 | $2,310 | 39.5% | | Total R&D expenses | $32,415 | $19,461 | $12,954 | 66.6% | [General and Administrative Expenses (Three Months)](index=31&type=section&id=General%20and%20Administrative%20Expenses%20(Three%20Months)) Analyzes the changes in general and administrative expenses for the three-month periods | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | General and administrative expenses | $9,278 | $8,327 | $951 | 11.4% | - The increase was primarily due to a **$1.2 million increase in personnel related costs**, partially offset by a **$0.3 million decrease in consulting fees**[125](index=125&type=chunk) [Other Income, Net (Three Months)](index=32&type=section&id=Other%20Income,%20Net%20(Three%20Months)) Reports the net other income and its components for the three-month periods | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :------------------------------------ | :------------------------------------ | :------- | :------- | | Total other income, net | $2,213 | $2,716 | $(503) | -18.5% | | Change in fair value of derivative liabilities | $(900) | $0 | $(900) | -100.0% | [Comparison of the Six Months Ended June 30, 2025 and 2024](index=32&type=section&id=Comparison%20of%20the%20Six%20Months%20Ended%20June%2030,%202025%20and%202024) Compares key financial metrics for the six-month periods, including operating expenses and net loss | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | Research and development | $63,329 | $38,334 | $24,995 | 65.2% | | General and administrative | $18,416 | $16,316 | $2,100 | 12.9% | | Total operating expenses | $81,745 | $54,650 | $27,095 | 49.6% | | Net loss | $(78,762) | $(49,375) | $(29,387) | 59.5% | [Research and Development Expenses (Six Months)](index=32&type=section&id=Research%20and%20Development%20Expenses%20(Six%20Months)) Details the changes in research and development expenses for the six-month periods | Candidate/Category | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | SGT-003 | $21,616 | $4,341 | $17,275 | 397.9% | | SGT-601 | $4,422 | $411 | $4,011 | 975.9% | | SGT-212 | $1,970 | $173 | $1,797 | 1038.7% | | Personnel related expenses (unallocated) | $16,150 | $11,917 | $4,233 | 35.5% | | Total R&D expenses | $63,329 | $38,334 | $24,995 | 65.2% | [General and Administrative Expenses (Six Months)](index=32&type=section&id=General%20and%20Administrative%20Expenses%20(Six%20Months)) Analyzes the changes in general and administrative expenses for the six-month periods | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | General and administrative expenses | $18,416 | $16,316 | $2,100 | 12.9% | - The increase was primarily due to a **$3.0 million increase in personnel related costs**, partially offset by a **$0.7 million decrease in legal and consulting fees**[129](index=129&type=chunk) [Other Income, Net (Six Months)](index=34&type=section&id=Other%20Income,%20Net%20(Six%20Months)) Reports the net other income and its components for the six-month periods | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | $ Change | % Change | | :-------------------------------- | :---------------------------------- | :---------------------------------- | :------- | :------- | | Total other income, net | $2,983 | $5,275 | $(2,292) | -43.5% | | Change in fair value of derivative liabilities | $(2,550) | $0 | $(2,550) | -100.0% | [Liquidity and Capital Resources](index=34&type=section&id=Liquidity%20and%20Capital%20Resources) Discusses the company's cash position, funding sources, and future capital requirements [Sources of Liquidity](index=34&type=section&id=Sources%20of%20Liquidity) Identifies the primary sources of the company's capital, including equity offerings - The company has raised an aggregate of **$857.1 million in net proceeds** from common stock sales through public offerings, private placements, and ATM sales agreements[131](index=131&type=chunk) - A February 2025 public offering generated approximately **$188.0 million in net proceeds** from the sale of common stock and pre-funded warrants[134](index=134&type=chunk) - As of June 30, 2025, the company had **$268.1 million in cash, cash equivalents, and available-for-sale securities** (excluding restricted cash) and no outstanding debt[135](index=135&type=chunk) [Summary of Cash Flows](index=34&type=section&id=Summary%20of%20Cash%20Flows) Provides a summary of cash flows from operating, investing, and financing activities | Cash Flow Activity | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------------------- | :------------------------------------ | :------------------------------------ | | Cash used in operating activities | $(69,262) | $(46,659) | | Cash used in investing activities | $(60,195) | $(43,666) | | Cash provided by financing activities | $188,127 | $112,241 | | Net increase in cash, cash equivalents, and restricted cash | $58,670 | $21,916 | [Operating Activities](index=34&type=section&id=Operating%20Activities) Analyzes the net cash used in operating activities and its primary drivers - Net cash used in operating activities was **$69.3 million** for the six months ended June 30, 2025, compared to **$46.7 million** for the same period in 2024[137](index=137&type=chunk)[138](index=138&type=chunk) - The increase in cash used was primarily due to a net loss of **$78.8 million** in H1 2025, partially offset by **$10.2 million in non-cash charges** (equity-based compensation, derivative liabilities, non-cash lease expense, depreciation/amortization)[137](index=137&type=chunk) [Investing Activities](index=36&type=section&id=Investing%20Activities) Details the net cash used in investing activities, primarily related to securities purchases and maturities - Net cash used in investing activities was **$60.2 million** for the six months ended June 30, 2025, compared to **$43.7 million** for the same period in 2024[139](index=139&type=chunk)[140](index=140&type=chunk) - This was primarily due to **$128.8 million in purchases of available-for-sale securities**, partially offset by **$69.0 million from maturities** of available-for-sale securities in H1 2025[139](index=139&type=chunk) [Financing Activities](index=36&type=section&id=Financing%20Activities) Explains the net cash provided by financing activities, mainly from public offerings - Net cash provided by financing activities was **$188.1 million** for the six months ended June 30, 2025, compared to **$112.2 million** for the same period in 2024[141](index=141&type=chunk)[142](index=142&type=chunk) - The increase was primarily driven by **$189.6 million in net proceeds** from the issuance and sale of common stock and pre-funded warrants in a public offering in H1 2025[141](index=141&type=chunk) [Funding Requirements](index=36&type=section&id=Funding%20Requirements) Outlines anticipated increases in expenses and the need for additional capital beyond current projections - Expenses are expected to increase substantially due to advancing clinical development of SGT-003, initiating clinical trials for SGT-212 and SGT-501, and other R&D activities[143](index=143&type=chunk) - As of June 30, 2025, cash, cash equivalents, and available-for-sale securities of **$268.1 million** are expected to fund operations into the first half of 2027[144](index=144&type=chunk) - The company will need to raise additional funds to operate beyond the first half of 2027, with future capital requirements dependent on clinical trial progress, regulatory outcomes, manufacturing costs, and potential collaborations[144](index=144&type=chunk)[145](index=145&type=chunk) [Contractual Obligations and Commitments](index=38&type=section&id=Contractual%20Obligations%20and%20Commitments) States that no material changes occurred in contractual obligations during the reporting period - No material changes to contractual obligations and commitments occurred during the six months ended June 30, 2025[151](index=151&type=chunk) [Recently Issued Accounting Pronouncements](index=38&type=section&id=Recently%20Issued%20Accounting%20Pronouncements) Discusses new accounting standards and their potential impact on financial statements and disclosures - ASU 2023-09 (Income Taxes) is effective for annual periods beginning after December 15, 2024, requiring greater disaggregation of income tax disclosures[44](index=44&type=chunk) - ASU 2024-03 (Disaggregation of Income Statement Expenses) is effective for annual periods beginning after December 15, 2026, requiring additional disclosures about specific expense categories[45](index=45&type=chunk) - The company is currently evaluating the impact of adopting these ASUs on its consolidated financial statements and disclosures[44](index=44&type=chunk)[45](index=45&type=chunk) [Off-Balance Sheet Arrangements](index=38&type=section&id=Off-Balance%20Sheet%20Arrangements) Confirms the absence of any off-balance sheet arrangements during the reported periods - The company did not have any off-balance sheet arrangements during the periods presented[153](index=153&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=38&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Discusses the company's exposure to market risks, including interest rate and inflation risks, and their potential financial impact [Interest Rate Risk](index=38&type=section&id=Interest%20Rate%20Risk) Assesses the impact of changes in market interest rates on the company's investment portfolio - The company's cash equivalents and investments consist of money market funds, treasury bills, and government bonds with contractual maturities of less than 90 days and one year, respectively[154](index=154&type=chunk) - An immediate **10% change in market interest rates** would not have a material impact on the fair market value of the investment portfolio or financial results[154](index=154&type=chunk) [Inflation Risk](index=38&type=section&id=Inflation%20Risk) Evaluates the potential adverse effects of inflation on the company's operating costs and business - Inflation increases costs of labor, research, manufacturing, and development[155](index=155&type=chunk) - Inflation has not had a material effect on the company's business in the last two years, but future operations could be adversely affected[155](index=155&type=chunk) [Item 4. Controls and Procedures](index=38&type=section&id=Item%204.%20Controls%20and%20Procedures) Details the evaluation of disclosure controls and procedures, confirming their effectiveness and no material changes [Evaluation of Disclosure Controls and Procedures](index=38&type=section&id=Evaluation%20of%20Disclosure%20Controls%20and%20Procedures) Reports management's assessment of the effectiveness of disclosure controls and procedures - Management, with CEO and CFO participation, evaluated disclosure controls and procedures as of June 30, 2025[156](index=156&type=chunk) - Disclosure controls and procedures were concluded to be designed and operating effectively, providing reasonable assurance[157](index=157&type=chunk) - Management acknowledges that control systems provide reasonable, not absolute, assurance and involve judgment in cost-benefit evaluation[157](index=157&type=chunk) [Changes in Internal Control over Financial Reporting](index=40&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) Confirms no material changes in internal control over financial reporting during the quarter - No material changes in internal control over financial reporting occurred during the three months ended June 30, 2025[158](index=158&type=chunk) [PART II. OTHER INFORMATION](index=41&type=section&id=PART%20II.%20OTHER%20INFORMATION) This part covers legal proceedings, comprehensive risk factors, equity security sales, and other relevant disclosures [Item 1. Legal Proceedings](index=41&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the company is not currently involved in any material legal proceedings - No legal proceedings were reported[160](index=160&type=chunk) [Item 1A. Risk Factors](index=41&type=section&id=Item%201A.%20Risk%20Factors) Outlines various risks that could materially affect the company's business, financial condition, and stock price [RISK FACTOR SUMMARY](index=41&type=section&id=RISK%20FACTOR%20SUMMARY) Summarizes the key financial, developmental, manufacturing, and intellectual property risks facing the company - The company has incurred significant net losses since inception and anticipates continued losses, requiring additional funding which may not be available on acceptable terms[163](index=163&type=chunk) - Key risks include the novel technology of gene transfer candidates, potential for clinical holds or adverse events, difficulty in enrolling participants, and challenges in obtaining regulatory approval[163](index=163&type=chunk) - Manufacturing limitations, reliance on third parties, competitive landscape, and intellectual property protection are also significant risk factors[163](index=163&type=chunk)[165](index=165&type=chunk) [Risks Related to our Financial Position and Need for Capital Requirements](index=43&type=section&id=Risks%20Related%20to%20our%20Financial%20Position%20and%20Need%20for%20Capital%20Requirements) Highlights risks associated with ongoing losses, future funding needs, and potential dilution or restrictive covenants - The company had a net loss of **$78.8 million** for the six months ended June 30, 2025, and an accumulated deficit of **$862.2 million**, expecting significant expenses and operating losses for the foreseeable future[164](index=164&type=chunk)[106](index=106&type=chunk) - Current cash, cash equivalents, and available-for-sale securities are projected to fund operations into the first half of 2027, but substantial additional funding will be needed thereafter[168](index=168&type=chunk)[109](index=109&type=chunk) - Failure to obtain additional capital could force delays, reductions, or termination of product development efforts, and raising capital may cause dilution to existing stockholders or involve restrictive debt covenants[168](index=168&type=chunk)[172](index=172&type=chunk) [Risks Related to the Development of our Candidates](index=50&type=section&id=Risks%20Related%20to%20the%20Development%20of%20our%20Candidates) Addresses challenges in gene therapy development, including clinical trial failures, adverse effects, and enrollment difficulties - Development of novel gene therapy candidates is difficult, with unpredictable time and cost, and a high risk of failure due to potential toxicity, lack of efficacy, or unforeseen issues[182](index=182&type=chunk) - Candidates may cause undesirable side effects, leading to clinical trial delays, regulatory approval denial, or withdrawal, as seen with past serious adverse events in gene therapy[185](index=185&type=chunk)[186](index=186&type=chunk)[187](index=187&type=chunk) - Difficulties in enrolling participants, especially for rare diseases, and potential non-acceptance of foreign clinical trial data by the FDA could delay or prevent clinical trials and regulatory approval[203](index=203&type=chunk)[206](index=206&type=chunk) [Risks Related to the Manufacturing and Commercialization of our Candidates](index=77&type=section&id=Risks%20Related%20to%20the%20Manufacturing%20and%20Commercialization%20of%20our%20Candidates) Covers risks in manufacturing, market acceptance, reimbursement, and competition for gene therapy products - The company has limited gene therapy manufacturing experience and relies on third-party manufacturers, facing risks of production problems, delays, and non-compliance with cGMP requirements[285](index=285&type=chunk)[293](index=293&type=chunk) - Commercial success depends on market acceptance by physicians, patients, and third-party payors, which is uncertain for novel gene therapy products due to ethical, social, medical, and legal concerns[303](index=303&type=chunk)[306](index=306&type=chunk) - Obtaining adequate insurance coverage and reimbursement for high-cost gene therapies is crucial but uncertain, with governments outside the U.S. often imposing strict price controls[311](index=311&type=chunk)[313](index=313&type=chunk)[314](index=314&type=chunk) [Risks Related to our Business Operations](index=89&type=section&id=Risks%20Related%20to%20our%20Business%20Operations) Examines operational risks including personnel retention, regulatory compliance, and changes in healthcare laws - The company's success depends on retaining key employees and attracting qualified personnel, facing intense competition in the gene therapy field[322](index=322&type=chunk)[323](index=323&type=chunk) - Operations are subject to federal and state healthcare fraud and abuse laws, false claims laws, and privacy/security laws (HIPAA, CCPA, GDPR), with potential for significant fines, penalties, and reputational damage for non-compliance[346](index=346&type=chunk)[350](index=350&type=chunk)[358](index=358&type=chunk) - Changes in tax laws (e.g., IRA, OBBBA) and legislative efforts to limit drug prices (e.g., Medicare price negotiations) could adversely affect the company's business and financial condition[331](index=331&type=chunk)[336](index=336&type=chunk)[337](index=337&type=chunk) [Risks Related to our Intellectual Property](index=105&type=section&id=Risks%20Related%20to%20our%20Intellectual%20Property) Details risks concerning patent protection, in-licensed rights, and the potential for intellectual property disputes - The company's ability to develop candidates depends heavily on in-licensed patent rights, with risks of license termination or inability to obtain additional necessary licenses on reasonable terms[378](index=378&type=chunk)[380](index=380&type=chunk) - Patent protection is highly uncertain; pending applications may not issue, issued patents may be challenged, narrowed, or invalidated, and competitors may design around patents[390](index=390&type=chunk)[392](index=392&type=chunk)[394](index=394&type=chunk) - Failure to protect trade secrets or disputes over intellectual property ownership could harm the business and competitive position[420](index=420&type=chunk)[424](index=424&type=chunk) [Risks Related to Ownership of our Common Stock](index=125&type=section&id=Risks%20Related%20to%20Ownership%20of%20our%20Common%20Stock) Addresses risks related to stock ownership, including control by insiders, potential dilution, and stock price volatility - Executive officers, directors, and principal stockholders collectively own a significant percentage of capital stock, enabling them to control or significantly influence matters submitted to stockholders[439](index=439&type=chunk) - Sales of a substantial number of common stock shares in the near future, including those from private placements and equity compensation plans, could cause the market price to drop significantly[440](index=440&type=chunk)[445](index=445&type=chunk)[446](index=446&type=chunk) - The company's stock price has been and is likely to remain volatile, influenced by clinical trial results, regulatory developments, competition, and financial performance, potentially leading to substantial losses for stockholders[448](index=448&type=chunk)[449](index=449&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=131&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section confirms that the company did not engage in any unregistered sales of equity securities during the three months ended June 30, 2025, that have not been previously disclosed - No unregistered securities were sold during the three months ended June 30, 2025, that were not previously described[462](index=462&type=chunk) [Item 5. Other Information](index=131&type=section&id=Item%205.%20Other%20Information) This section reports that no directors or officers adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the second quarter of 2025 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or officers during Q2 2025[463](index=463&type=chunk) [Item 6. Exhibits](index=132&type=section&id=Item%206.%20Exhibits) Lists the exhibits filed with the Form 10-Q, including corporate documents and officer certifications | Exhibit Number | Description | | :------------- | :---------- | | 3.1 | Certificate of Incorporation of Solid Biosciences Inc., as amended | | 10.1 | Amended and Restated 2020 Equity Incentive Plan, as amended | | 31.1* | Certification of Principal Executive Officer Pursuant to Rules 13a-14(a) and 15d-14(a) | | 31.2* | Certification of Principal Financial Officer Pursuant to Rules 13a-14(a) and 15d-14(a) | | 32.1** | Certification of Principal Executive Officer Pursuant to 18 U.S.C. Section 1350 | | 32.2** | Certification of Principal Financial Officer Pursuant to 18 U.S.C. Section 1350 | | 101.INS | Inline XBRL Instance Document | | 101.SCH | Inline XBRL Taxonomy Extension Schema Document | | 104 | Cover Page Interactive Data File | [SIGNATURES](index=133&type=section&id=SIGNATURES) This section contains the official signatures of the principal executive and financial officers, certifying the report's submission [Signatures](index=133&type=section&id=Signatures) The report is signed by Alexander Cumbo, President and Chief Executive Officer, and Kevin Tan, Chief Financial Officer, on August 12, 2025, certifying its submission - The report was signed by Alexander Cumbo (President and CEO) and Kevin Tan (CFO) on August 12, 2025[470](index=470&type=chunk)