This presentation release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company's priorities and achieve key clinical milestones; the company's plans to present data from its Friedreich's ataxia program, next-generation Duchenne muscular dystrophy program, novel capsid program, and process developme ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) (State or other jurisdict ...

Financial Performance - The company incurred a net loss of $86.0 million for the year ended December 31, 2022, with an accumulated deficit of $562.7 million as of the same date [237]. - The company expects to continue incurring significant operating losses for the foreseeable future as it focuses on research and development [237]. - The company has never generated revenue from product sales and does not expect to do so for the foreseeable future [244]. - The company will need to raise additional funds to continue operations beyond 2025, with no assurance that such funds will be available on acceptable terms [240]. Acquisition and Integration - The acquisition of AavantiBio was completed on December 2, 2022, and included pipeline programs for treating FA and BAG3 mediated dilated cardiomyopathy [230]. - There is a risk that the integration of AavantiBio may divert management's attention and disrupt ongoing business operations [231]. - The anticipated benefits of the acquisition may not be realized if integration challenges arise or if undisclosed liabilities surface [232]. - Future capital requirements will depend on the progress and costs associated with integrating AavantiBio and the results of clinical trials for SGT-003 and other candidates [241]. - The company may face increased litigation risks following the acquisition, which could adversely impact business operations [236]. Clinical Trials and Regulatory Challenges - The COVID-19 pandemic has caused disruptions in clinical trials, regulatory activities, and supply chains, impacting the ability to complete preclinical studies and clinical trials [247][248]. - Company has faced challenges in recruiting patients for clinical trials due to COVID-19, resulting in missed or postponed patient visits [249]. - Regulatory approvals may be delayed or impacted due to the redirection of resources by the FDA towards COVID-19 therapies, affecting the progress of the company's product candidates [250]. - The company has limited experience in regulatory submissions and has never completed a clinical trial, which poses risks for future product candidates [272]. - The company may face significant delays in clinical trials due to various factors, including patient recruitment and regulatory approvals [278]. - Regulatory approval for SGT-003, AVB-202-TT, and AVB-401 is uncertain, and even if trials are successful, approvals may be for narrower indications than sought [285]. Product Development and Safety Concerns - The regulatory approval process for gene transfer candidates is uncertain and can be more expensive and time-consuming compared to other products, with only a limited number of such products approved in the U.S. and EU [262]. - There is a risk of undesirable side effects from gene transfer candidates that could delay or prevent regulatory approval, impacting their commercial potential [263]. - Previous gene therapy treatments have reported significant adverse side effects, raising concerns about the safety and efficacy of the company's candidates [266]. - A serious adverse event related to SGT-001 occurred in a clinical trial, leading to a clinical hold by the FDA in November 2019, which was lifted in October 2020 [270]. - The anticipated dosing requirements for SGT-003 may increase the risk of adverse side effects, as seen in previous high-dose AAV vector administrations [268]. Manufacturing and Supply Chain Risks - The company currently relies on third-party manufacturers for SGT-003 and plans to do so for AVB-202-TT and AVB-401 programs, which exposes it to risks of delays and insufficient supply [351]. - The company does not have long-term supply arrangements for SGT-003, which may hinder its ability to scale production and meet future demand [353]. - There is a risk of significant disruption in supply due to factors such as equipment malfunctions, contamination, or public health issues like the COVID-19 pandemic [346]. - The company is at risk of contamination in its manufacturing processes, which could disrupt the supply of its product candidates and affect clinical development timelines [366]. Market and Competitive Landscape - The company faces significant competition from larger pharmaceutical and biotechnology firms, which may achieve regulatory approval before the company or develop more effective therapies [328]. - Competitors such as Pfizer and Sarepta Therapeutics are advancing in clinical trials for gene therapies, with Sarepta having submitted a BLA for its candidate with a PDUFA date of May 29, 2023 [329]. - The company's commercial opportunity may be diminished if competitors launch safer, more effective, or less expensive products first [330]. - The commercial success of gene therapy products may be limited by patients' immune responses, affecting the population amenable to treatment [360]. Regulatory Environment and Compliance - The FDA may require a Risk Evaluation and Mitigation Strategy (REMS) if SGT-003 or other candidates receive marketing approval, which could include a medication guide and communication plan [269]. - Compliance with ongoing regulatory requirements post-approval is necessary, and failure to do so could limit the ability to market future products [291]. - The company is subject to various stringent privacy laws and regulations, including HIPAA, which impose significant compliance costs and potential penalties for violations [405]. - The company faces risks related to anti-corruption laws, including the U.K. Bribery Act and the U.S. Foreign Corrupt Practices Act, which could lead to civil or criminal penalties if violated [416]. Financial and Operational Risks - The company faces potential pricing pressures from both federal and state healthcare reforms, which could limit revenue generation capabilities [396]. - The company has reduced its workforce by approximately 35% in April 2022 and 18% in December 2022 as part of a strategic plan to streamline operations, but may not realize the anticipated benefits from these reductions [381]. - The company is highly dependent on key employees, and the loss of these individuals could impede the achievement of its research and commercialization objectives [378]. - The company may engage in future acquisitions or strategic collaborations, which could increase capital requirements and introduce additional risks [376].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Sec ...

© 2022 Solid Biosciences 1 Strategic Update: Acquisition of AavantiBio and $75M PIPE September 30, 2022 Forward-Looking Statements and Industry and Market Data This presentation and various remarks we make during this presentation contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding: future expectations, plans and prospects for Solid Biosciences Inc. (the "Company" or "Solid"), AavantiBio, Inc. ("AavantiBio") and the ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 90-0943402 (State ...

Solid Biosciences Inc. (NASDAQ:SLDB) Q1 2022 Results Conference Call April 27, 2022 8:00 AM ET Company Participants Caitlin Lowie - VP, Communications and IR Ilan Ganot - Co-Founder, President and CEO Dr. Joel Schneider - COO Dr. Carl Morris - Chief Scientific Officer Dr. Roxana Donisa Dreghici - SVP and Head, Clinical Development Conference Call Participants Gena Wang - Barclays Joseph Schwartz - SVB Securities Allison Bratzel - Piper Sandler Anupam Rama - JP Morgan Maury Raycroft - Jefferies Operator Ladi ...

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Q1 2022 net loss of $25.3 million, decreased assets, and April 2022 restructuring with 35% workforce reduction Condensed Consolidated Balance Sheet Data (in thousands) | Account | March 31, 2022 | December 31, 2021 | | :--- | :--- | :--- | | Cash and cash equivalents | $129,711 | $119,136 | | Available-for-sale securities | $50,339 | $88,643 | | **Total Assets** | **$207,556** | **$232,380** | | Total current liabilities | $21,744 | $23,601 | | **Total Liabilities** | **$22,045** | **$24,169** | | **Total Stockholders' Equity** | **$185,511** | **$208,211** | Condensed Consolidated Statements of Operations (in thousands) | Account | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Collaboration revenue - related party | $1,925 | $3,335 | | Research and development | $19,945 | $14,206 | | General and administrative | $7,352 | $6,015 | | **Loss from operations** | **($25,372)** | **($16,886)** | | **Net loss** | **($25,328)** | **($16,900)** | | **Net loss per share** | **($0.22)** | **($0.19)** | Condensed Consolidated Statements of Cash Flows (in thousands) | Activity | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | ($27,190) | ($21,366) | | Net cash provided by (used in) investing activities | $37,743 | ($35) | | Net cash provided by financing activities | $22 | $135,154 | - In April 2022, the company implemented a corporate restructuring to prioritize its key programs, SGT-001 and SGT-003, involving a headcount reduction of approximately **35%**, with estimated restructuring costs of **$1.7 million** for severance and employee benefits[83](index=83&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=19&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses strategic shifts, including a 35% workforce reduction to extend cash runway, and Q1 2022 net loss of $25.3 million from increased R&D - The company's lead product candidate is **SGT-001**, a gene transfer therapy for Duchenne, with enrollment in the Phase I/II **IGNITE DMD** trial complete and two-year data suggesting **durable benefit**[90](index=90&type=chunk)[91](index=91&type=chunk)[94](index=94&type=chunk) - In **April 2022**, the company announced a **strategic shift** to a commercially scaled, transient transfection-based manufacturing process for **SGT-001** and narrowed its R&D focus to **SGT-001**, **SGT-003**, and next-generation capsids[92](index=92&type=chunk) - As part of the **April 2022** reorganization, the company reduced its headcount by approximately **35%**, expected to extend the cash runway into the **second quarter of 2024**, based on **$180.1 million** in cash, cash equivalents, and available-for-sale securities as of March 31, 2022[93](index=93&type=chunk)[102](index=102&type=chunk) Comparison of Operating Results (in thousands) | Line Item | Q1 2022 | Q1 2021 | Change | % Change | | :--- | :--- | :--- | :--- | :--- | | Collaboration Revenue | $1,925 | $3,335 | ($1,410) | (42%) | | R&D Expenses | $19,945 | $14,206 | $5,739 | 40% | | G&A Expenses | $7,352 | $6,015 | $1,337 | 22% | | **Net Loss** | **($25,328)** | **($16,900)** | **($8,428)** | **50%** | Research & Development Expenses Breakdown (in thousands) | Program | Q1 2022 | Q1 2021 | Change | % Change | | :--- | :--- | :--- | :--- | :--- | | SGT-001 | $5,422 | $6,173 | ($751) | (12)% | | SGT-003 and other programs | $3,364 | $192 | $3,172 | 1652% | | Unallocated R&D | $11,159 | $7,841 | $3,318 | 42% | | **Total R&D Expenses** | **$19,945** | **$14,206** | **$5,739** | **40%** | [Quantitative and Qualitative Disclosures About Market Risk](index=29&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces market risk from interest rate changes on short-term investments, with a 10% change not materially impacting financial position - The company's primary market risk exposure is **interest income sensitivity** related to its cash equivalents and available-for-sale securities[145](index=145&type=chunk) - Due to the short-term nature of its investment portfolio, a **10%** change in interest rates is **not expected to materially impact** the company's financial condition[145](index=145&type=chunk) [Controls and Procedures](index=29&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of March 31, 2022, with no material changes to internal control over financial reporting - As of March 31, 2022, the President and Chief Executive Officer and the interim Chief Financial Officer concluded that the company's disclosure controls and procedures were **effective at the reasonable assurance level**[146](index=146&type=chunk) - **No material changes** to the company's internal control over financial reporting occurred during the first quarter of 2022[147](index=147&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=30&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not a party to any material legal proceedings - As of the filing date, the company states there are **no material legal proceedings**[150](index=150&type=chunk) [Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant financial, developmental, manufacturing, commercial, and IP risks, including a history of losses and novel gene therapies - **Financial Risks:** The company has a history of significant net losses (**$25.3M** in Q1 2022) and an accumulated deficit of **$502.1M**, and will **require additional funding** to continue operations beyond its current cash runway[154](index=154&type=chunk)[158](index=158&type=chunk) - **Clinical Development Risks:** SGT-001 and SGT-003 are based on **novel gene transfer technology**, making development time and cost difficult to predict, and the IGNITE DMD trial was previously placed on **clinical hold** by the FDA[153](index=153&type=chunk)[172](index=172&type=chunk)[175](index=175&type=chunk) - **Manufacturing & Commercialization Risks:** The company has **limited gene transfer manufacturing experience** and **relies on third parties**, posing risks of performance, regulatory compliance, and **supply disruption**, with a planned shift to a new manufacturing process for SGT-001 potentially requiring additional studies[155](index=155&type=chunk)[249](index=249&type=chunk)[256](index=256&type=chunk) - **Intellectual Property Risks:** The business **heavily relies on in-licensed patents**, which the company may **not control the prosecution and enforcement** of, and they could be **challenged, invalidated, or circumvented** by competitors[155](index=155&type=chunk)[328](index=328&type=chunk)[334](index=334&type=chunk) - **Competition Risk:** The company faces **significant competition** from **larger, better-funded companies** like Pfizer and Sarepta Therapeutics, which have Duchenne gene therapy candidates in **later stages of clinical development (Phase III)**[235](index=235&type=chunk)[236](index=236&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=88&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) On January 3, 2022, the company issued an inducement grant of 131,400 stock options and 65,700 restricted stock units - On **January 3, 2022**, the company granted a new employee an option for **131,400 shares** and **65,700 restricted stock units** as an **inducement award** under Nasdaq Listing Rule 5635(c)(4)[403](index=403&type=chunk) [Other Information](index=88&type=section&id=Item%205.%20Other%20Information) On April 25, 2022, the Board approved a restructuring plan with a 35% workforce reduction, costing $1.7 million, to extend cash runway into Q2 2024 - On **April 25, 2022**, the Board approved a restructuring plan to reduce the workforce by approximately **35%** to prioritize key programs and extend the cash runway into **Q2 2024**[405](index=405&type=chunk)[406](index=406&type=chunk) - The company estimates total restructuring costs of approximately **$1.7 million**, primarily for severance and employee termination benefits, to be paid mostly during 2022[407](index=407&type=chunk) - A **retention program** was approved for remaining key employees, including cash bonuses and stock option grants for the Chief Legal Officer and Chief Scientific Officer[408](index=408&type=chunk) [Exhibits](index=89&type=section&id=Item%206.%20Exhibits) The section lists exhibits filed with Form 10-Q, including CEO/CFO certifications and Inline XBRL documents for financial reporting - The filing includes **CEO and CFO certifications** pursuant to Sections 302 and 906 of the Sarbanes-Oxley Act[410](index=410&type=chunk) - **Inline XBRL** Instance, Schema, and other related taxonomy documents are included as exhibits for interactive data[410](index=410&type=chunk) [Signatures](index=90&type=section&id=Signatures) The report was signed on April 27, 2022, by Ilan Ganot (CEO) and Stephen DiPalma (Interim CFO) - The report was signed on **April 27, 2022**, by **Ilan Ganot (CEO)** and **Stephen DiPalma (Interim CFO)**[413](index=413&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number 001-38360 Solid Biosciences Inc. (Exact name of Registrant as specified in its Charter) (State or other jurisdiction of incorporation or ...

[PART I. FINANCIAL INFORMATION](index=2&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=2&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents unaudited financial statements reflecting increased equity from a public offering and new collaboration revenue [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet reflects a substantial increase in total assets and stockholders' equity, driven by a public offering Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2021 | Dec 31, 2020 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $109,676 | $154,744 | | Available-for-sale securities | $120,084 | $— | | **Total current assets** | **$238,810** | **$158,901** | | **Total assets** | **$248,986** | **$171,169** | | **Liabilities & Equity** | | | | Total current liabilities | $22,542 | $24,480 | | **Total liabilities** | **$25,623** | **$39,083** | | **Total stockholders' equity** | **$223,363** | **$132,086** | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Operations show new collaboration revenue and reduced R&D expenses, resulting in a lower net loss compared to the prior year Statement of Operations Summary (in thousands, except per share data) | Metric | Q3 2021 | Q3 2020 | 9 Months 2021 | 9 Months 2020 | | :--- | :--- | :--- | :--- | :--- | | Collaboration revenue | $3,537 | $— | $10,466 | $— | | Research and development | $14,425 | $16,045 | $44,144 | $49,158 | | General and administrative | $7,143 | $5,181 | $19,924 | $15,957 | | Loss from operations | $(18,031) | $(21,226) | $(53,602) | $(67,059) | | **Net loss** | **$(17,983)** | **$(21,246)** | **$(53,578)** | **$(66,927)** | | Net loss per share | $(0.16) | $(0.44) | $(0.51) | $(1.39) | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash flows indicate significant financing from a stock issuance, offset by cash used for investments and operations Cash Flow Summary (Nine Months Ended Sep 30, in thousands) | Cash Flow Activity | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(56,890) | $(57,906) | | Net cash (used in) provided by investing activities | $(121,262) | $6,660 | | Net cash provided by financing activities | $134,919 | $— | | **Net decrease in cash, cash equivalents and restricted cash** | **$(43,233)** | **$(51,246)** | [Notes to the Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20the%20Condensed%20Consolidated%20Financial%20Statements) Notes detail the IGNITE DMD trial status, the Ultragenyx collaboration, and the company's sufficient liquidity for the next twelve months - The company's lead product candidate, SGT-001, is in a Phase I/II clinical trial (IGNITE DMD) which was previously on clinical hold by the FDA and resumed patient treatment in February 2021[25](index=25&type=chunk) - The company believes its cash, cash equivalents, and available-for-sale securities of **$229.8 million** as of September 30, 2021, are sufficient to fund operations for at least twelve months[30](index=30&type=chunk) - The collaboration with Ultragenyx resulted in recognizing **$10.5 million** of related party collaboration revenue for the nine months ended September 30, 2021[61](index=61&type=chunk) - In March 2021, the company completed a public offering of 25,000,000 shares of common stock, receiving net proceeds of approximately **$134.9 million**[76](index=76&type=chunk)[109](index=109&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the clinical progress of SGT-001, financial performance improvements, and a cash runway into Q2 2023 [Overview](index=20&type=section&id=MD%26A%20Overview) The company focuses on its SGT-001 trial for Duchenne, manages a recent adverse event, and advances its next-gen SGT-003 candidate - The eighth patient in the IGNITE DMD trial experienced a **serious adverse event**, which has since fully resolved, and a strengthened risk mitigation plan has been submitted to the FDA[91](index=91&type=chunk)[92](index=92&type=chunk)[93](index=93&type=chunk) - Long-term biopsy data from patients 4-6 demonstrated **durable and widespread expression of microdystrophin protein** at 1 to 2 years post-dosing[95](index=95&type=chunk) - The company is advancing a next-generation program, SGT-003, and is targeting an **IND submission in early 2023**[99](index=99&type=chunk) - As of September 30, 2021, the company had cash, cash equivalents, and available-for-sale securities of **$229.8 million**, expected to fund operations into the second quarter of 2023[110](index=110&type=chunk) [Results of Operations](index=27&type=section&id=MD%26A%20Results%20of%20Operations) Financial results improved due to new collaboration revenue and lower R&D costs, despite an increase in G&A expenses Comparison of Results (in thousands) | Metric | 9 Months 2021 | 9 Months 2020 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $10,466 | $— | $10,466 | | Research & Development | $44,144 | $49,158 | $(5,014) | | General & Administrative | $19,924 | $15,957 | $3,967 | | **Net Loss** | **$(53,578)** | **$(66,927)** | **$13,349** | - The **$5.0 million decrease in R&D expenses** was primarily due to a $6.4 million decrease in SGT-001 manufacturing costs, partially offset by higher personnel expenses[137](index=137&type=chunk) - The **$3.9 million increase in G&A expenses** was due to a $2.2 million increase in corporate expenses and a $1.7 million increase in personnel-related expenses[138](index=138&type=chunk) [Liquidity and Capital Resources](index=28&type=section&id=MD%26A%20Liquidity%20and%20Capital%20Resources) The company's liquidity is strong following recent equity financing, providing a cash runway into the second quarter of 2023 - The company raised net proceeds of approximately **$134.9 million** from a public offering in March 2021[147](index=147&type=chunk) - As of September 30, 2021, the company had cash, cash equivalents and available-for-sale securities of **$229.8 million** and no debt outstanding[148](index=148&type=chunk) - The company expects its current cash position will be sufficient to fund operations into the **second quarter of 2023**, but will need to raise additional funds to operate beyond that time[155](index=155&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk from interest rate changes on its short-term investments is considered immaterial - The company's primary market risk is **interest rate sensitivity** on its cash equivalents and available-for-sale securities[165](index=165&type=chunk) - Due to the short-term nature of its investment portfolio, the company does not expect a 10% change in interest rates to have a **material impact** on its financial position[165](index=165&type=chunk) [Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective, with no material changes to internal controls - Management concluded that as of September 30, 2021, the company's disclosure controls and procedures were **effective at the reasonable assurance level**[166](index=166&type=chunk) - **No material changes** in internal control over financial reporting occurred during the three months ended September 30, 2021[167](index=167&type=chunk) [PART II. OTHER INFORMATION](index=32&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=34&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings - As of September 30, 2021, the company is **not aware of any material legal proceedings**[82](index=82&type=chunk)[170](index=170&type=chunk) [Risk Factors](index=34&type=section&id=Item%201A.%20Risk%20Factors) Key risks include a history of net losses, clinical development challenges, manufacturing reliance, and intellectual property dependencies - Financial risks include a history of significant net losses (**$458.1M accumulated deficit**) and the need for additional funding beyond Q2 2023[174](index=174&type=chunk)[177](index=177&type=chunk)[179](index=179&type=chunk) - **Clinical development risks are high**, highlighted by the previous FDA clinical hold on the IGNITE DMD trial for SGT-001 due to a serious adverse event[192](index=192&type=chunk) - The company has limited manufacturing experience and **relies on third parties**, facing risks of production problems, delays, and regulatory compliance issues[266](index=266&type=chunk) - The business is **heavily dependent on intellectual property licensed from universities**, which imposes diligence obligations and may be subject to government 'march-in' rights[347](index=347&type=chunk)[385](index=385&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=88&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company issued unregistered stock options as inducement awards to two new employees during the third quarter of 2021 - On July 1, 2021, granted an option to purchase **235,000 shares** at an exercise price of $3.77 per share to a new employee[424](index=424&type=chunk) - On August 16, 2021, granted an option to purchase **118,000 shares** at an exercise price of $2.51 per share to a new employee[425](index=425&type=chunk) [Exhibits](index=89&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the report, including stock plans, agreements, and officer certifications [Signatures](index=90&type=section&id=Signatures) The report is formally signed and authorized by the company's CEO and interim CFO