CHARLESTOWN, Mass., Feb. 06, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, will present at the Guggenheim Emerging Outlook: Biotech Summit 2026 on Thursday, February 12, 2026, at 10:30 AM ET. A live webcast of the presentation will be available on the Events page of the Investors section of the Company website or ...

Company Overview - Solid Biosciences has successfully dosed 33 patients with no reported drug-induced liver injury, myocarditis, or aHUS, indicating a strong safety profile for their treatments [2] - The company has enrolled its first patient in a double-blind, placebo-controlled study, marking a significant milestone in their clinical development [3] Business Development - Solid Biosciences has established 50 different agreements for their capsids, showcasing their growing reputation as a platform technology company [3] - The company is increasingly recognized for its delivery vehicles, which are being utilized in next-generation therapies, reflecting a positive trend in collaboration and innovation [3]

Summary of Solid Biosciences FY Conference Call Company Overview - **Company**: Solid Biosciences (NasdaqGS:SLDB) - **Event**: 44th Annual J.P. Morgan Healthcare Conference - **Date**: January 13, 2026 - **CEO**: Bo Cumbo Key Points Industry and Company Developments - Solid Biosciences has dosed 33 patients in its phase 1/2 Duchenne muscular dystrophy (DMD) trial called Inspire, reporting no drug-induced liver injury, myocarditis, or AHUS [2][30] - The company has successfully enrolled its first patient in a double-blind placebo-controlled trial and dosed its first patient in Friedreich's ataxia (FA) [2][5] - Solid has established over 50 partnerships for its capsid technology, indicating a growing interest in its platform for next-generation therapies [3][6] Clinical Trials and Pipeline - The Inspire trial aims to enroll 50 to 60 patients, with 33 already dosed and full enrollment expected soon [8][31] - The company is prioritizing FA over CPVT (catecholaminergic polymorphic ventricular tachycardia) due to resource constraints, with plans to dose patients in CPVT this quarter [5][6] - The first patient in the double-blind placebo-controlled trial will be dosed in either Australia or Canada, with European sites also opening [9][32] Safety and Efficacy Data - The safety profile of the therapy appears robust, with 0% drug-induced liver injury and other serious adverse events reported [11][30] - The mean expression of microdystrophin in treated patients is reported at 58%, the highest among current therapies [10][23] - Early cardiac data shows declines in troponin levels and increases in ejection fraction, suggesting potential cardiac benefits [11][28] Capsid Technology - The capsid SLB101 is designed for enhanced binding to muscle tissue, leading to improved distribution and reduced liver targeting [12][19] - The capsid technology is noted for its low viral load (1E14), which is lower than other therapies, potentially leading to fewer side effects [14][40] - The capsid's unique design allows for better muscle perfusion and reduced fibrosis, contributing to the observed clinical benefits [13][19] Market Opportunity - The DMD market is expanding, with approximately 100 boys born with the disease each quarter, indicating a high demand for new therapies [36] - Solid aims to be a leader in the DMD space, with plans for accelerated approval pathways and potential first gene therapy to market in the UK [36][44] Regulatory Strategy - The company is preparing for multiple meetings with the FDA to discuss the phase 3 placebo-controlled trial and the potential for accelerated approval [33][44] - Solid has not reviewed any functional data to maintain the integrity of its discussions with the FDA, ensuring confidence in its regulatory submissions [34][45] Future Milestones - Solid plans to continue dosing patients and gathering data throughout the year, with significant milestones expected that could transform the company [38][39] - The company is focused on ensuring that its therapies are well-positioned for market entry and regulatory approval, with ongoing studies to track long-term outcomes [29][44] Additional Insights - The dual-route administration in FA is a significant milestone, showcasing the company's innovative approach to treatment delivery [37] - The emphasis on safety and efficacy data, along with a strong pipeline, positions Solid Biosciences as a key player in the gene therapy landscape for muscular dystrophies [12][30]

Corporate Presentation January 2026 © 2026 Solid Biosciences Cautionary Note Regarding Forward Looking Statements This presentation 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 goals, priorities and key clinical and preclinical milestones; strategies and expectations for the company's SGT ...

Core Insights - Solid Biosciences is advancing its gene therapy programs for neuromuscular and cardiac diseases, with significant progress reported in multiple clinical trials as of January 2026 [1][2][3] Neuromuscular Pipeline - SGT-003 for Duchenne muscular dystrophy has been well tolerated in 33 participants dosed in the Phase 1/2 INSPIRE DUCHENNE trial, with no serious adverse events reported as of January 9, 2026 [3][4] - The company plans to engage with the FDA in the first half of 2026 to discuss the design of a Phase 3 confirmatory trial for SGT-003, aiming for potential accelerated approval [3][4] - The first participant has been enrolled in the Phase 3 IMPACT DUCHENNE trial, with dosing expected in Q1 2026 [1][7] - SGT-212 for Friedreich's ataxia has received FDA Orphan Drug designation and the first participant has been dosed in the Phase 1b FALCON trial, which is the first gene therapy for FA utilizing a dual route of administration [1][7] Cardiac Pipeline - SGT-501 is being evaluated in the ARTEMIS Phase 1b trial for catecholaminergic polymorphic ventricular tachycardia, with participant screening currently underway [1][7] - Initial safety data from the ARTEMIS trial is expected in the second half of 2026 [7] Platform Technologies - Solid Biosciences has executed over 50 agreements for the use of its proprietary next-generation capsid AAV-SLB101, which aims to enhance gene therapy delivery [1][5][7] - AAV-SLB101 has shown promising results in terms of safety and efficacy in the ongoing trials [7]

Core Insights - Solid Biosciences has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations for SGT-212, a gene therapy for Friedreich's ataxia (FA) [1][2] - The first participant has been dosed in the Phase 1b FALCON trial, with initial data expected in the second half of 2026 [1] Regulatory Designations - The Orphan Drug designation highlights the significant unmet need in the FA community and recognizes the therapeutic potential of SGT-212's dual-route administration [2] - These designations may help streamline and accelerate the development path for SGT-212 [2][3] FALCON Trial Details - FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial evaluating the safety and tolerability of SGT-212 in participants aged 18-40 diagnosed with FA and cardiac hypertrophy [4] - The trial is being conducted in the United States [4] SGT-212 Overview - SGT-212 is a recombinant AAV-based gene replacement therapy designed to deliver full-length human frataxin via a dual route: intradentate nucleus infusion followed by intravenous infusion [5] - The therapy aims to restore therapeutic levels of frataxin to address the neurological, cardiac, and systemic manifestations of FA [5] Friedreich's Ataxia (FA) Background - FA is a life-threatening, degenerative multisystem disease affecting approximately 5,000 people in the U.S. and 15,000 in Europe, with no current treatments available to cure or halt disease progression [6]

Core Viewpoint - Solid Biosciences Inc. has initiated dosing in the FALCON trial, a Phase 1b clinical trial for SGT-212, a gene therapy aimed at treating Friedreich's ataxia (FA) [1][3] Group 1: SGT-212 and FALCON Trial - SGT-212 is a first-in-class gene therapy designed to address the neurological, cardiac, and systemic manifestations of FA through a dual-route administration [2][5] - The FALCON trial is an open-label, multi-center study evaluating the safety and tolerability of SGT-212 in participants aged 18-40 diagnosed with FA and cardiac hypertrophy [4] - The therapy aims to restore therapeutic levels of frataxin, which is crucial for mitochondrial function and overall cellular health [5][6] Group 2: Company Statements and Future Outlook - The Chief Medical Officer of Solid Biosciences expressed confidence in the precision of the MRI-guided delivery method, which exceeded expectations for targeting the intradentate nuclei [3] - The President & CEO highlighted the significance of the trial for the FA community and the company's commitment to addressing both quality of life and mortality drivers associated with the disease [3] - Preliminary safety insights are expected to be shared in the coming months, with an initial data update anticipated in the second half of 2026 [3]

CHARLESTOWN, Mass., Jan. 06, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, will present at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 5:15 p.m. PT (8:15 p.m. ET). A live webcast of the presentation will be available on the Events page of the Investors section of the Company ...

Summary of Key Points from Biotechnology Equity Research Conference Call Industry Overview - The report focuses on the biotechnology sector, specifically highlighting companies with significant potential for growth and upcoming catalysts in 2026. Key Companies and Their Outlook 1. **Dianthus Therapeutics, Inc. (DNTH)** - Market Cap: $1.82 billion - Price Target: $66 - Key catalysts include: - CIDP Phase III interim analysis in Q2 2026 - Initiation of gMG Phase III study in mid-2026 - Phase II MMN topline data in 2H26 - Potential for significant upside based on peak revenue estimates of ~$2 billion across neuromuscular diseases [19][20][24] 2. **Taysha Gene Therapies, Inc. (TSHA)** - Market Cap: $1.50 billion - Price Target: $11 - Focus on pivotal study for TSHA-102 in Rett syndrome with potential for a 6-month interim readout by YE26/1Q27 [26][27] 3. **Tyra Biosciences (TYRA)** - Market Cap: $1.36 billion - Price Target: $32 - Major catalysts include: - Phase III data for achondroplasia in early 2026 - Initial data for IR-NMIBC in 1H26 - Potential to disrupt the market with oral FGFR3 inhibitors [31][35] 4. **Tango Therapeutics, Inc. (TNGX)** - Market Cap: $1.15 billion - Price Target: $14 - Expected to show de-risking data for vopimetostat in combination with RVMD's RAS inhibitors in 2026 [37][40] 5. **ORIC Pharmaceuticals (ORIC)** - Market Cap: $783 million - Price Target: $23 - Key catalysts include dose-optimization data for prostate cancer and validating data from PFE's MEVPRO-1 trial [42][48] 6. **Solid Biosciences (SLDB)** - Market Cap: $463 million - Price Target: $15 - Focus on DMD program with key regulatory feedback expected in 1H26 [49][53] Major Catalysts and Events - **Upcoming Catalysts:** - RARE and MREO Phase III ORBIT final analysis expected in Dec'25/Jan'26 with potential stock movements of +100%/-30% [2] - ALNY, CRSP, and others expected to provide revenue guidance and business plans early in 2026 [1] - Regulatory events including FDA's Rare Disease Day on 2/23/26 [12] - **Drug Launches:** - Expected launches in 2026 include drugs from ALNY, ARWR, KALV, and others targeting various conditions [3] Financial Updates - **Price Target Changes:** - ARWR raised from $67 to $90 - KOD raised from $24 to $39 [6][10] Market Dynamics - **Pricing Stability:** - 2026 net pricing expected to remain stable with current Medicare contracts influencing high gross-to-net (GTN) guidance [12] - **Strategic Interest:** - Potential for M&A activity as companies like ORIC attract interest due to their prostate cancer programs [43] Conclusion - The biotechnology sector is poised for significant developments in 2026, with multiple companies presenting strong investment opportunities based on upcoming catalysts, drug launches, and strategic positioning in the market.

Core Insights - Jefferies has released a report on the outlook for the U.S. biotech industry in 2026, highlighting key clinical trial data disclosures, new drug approvals, and policy dynamics that could lead to value re-evaluation for companies in the sector [1] Six Preferred Stocks - Jefferies identified six companies as core recommendations for 2026 based on market catalysts and valuation potential: - Dianthus Therapeutics (DNTH.US), target price $66: Focused on claseprubart for various neuromuscular diseases, with peak revenue estimated at $2 billion [2] - Taysha Gene Therapies (TSHA.US), target price $11: TSHA-102 for Rett syndrome, with peak revenue potential of $2 billion and a key trial data disclosure expected by the end of 2026 [2] - Tyra Biosciences (TYRA.US), target price $32: Dabogratinib for bladder cancer and achondroplasia, with critical data expected next year [3] - Tango Therapeutics (TNGX.US), target price $14: Collaboration with Revolution Medicines for a pancreatic cancer treatment, with data expected in mid-2026 [3] - ORIC Pharmaceuticals (ORIC.US), target price $23: Two potential assets targeting prostate cancer and lung cancer, with key data expected next year [4] - Solid Biosciences (SLDB.US), target price $15: Focused on gene therapy for Duchenne muscular dystrophy, with critical meetings with the FDA planned for 2026 [4] Seven Undervalued Companies - The report also highlighted seven undervalued companies with significant catalysts: - Ultragenyx Pharmaceutical (RARE.US), target price $114: Expected key catalysts in 2026, including data for treatments for osteogenesis imperfecta and Angelman syndrome [5] - Beam Therapeutics (BEAM.US), target price $41: Updates on AATD therapy and SCD therapy expected in 2026, showcasing strong potential [5] - Aurinia Pharmaceuticals (AUPH.US), target price $21: Steady growth in lupus nephritis treatment, with new drug development paths expected to expand growth [6] - Kodiak Sciences (KOD.US), target price $39: Anticipated data from three pivotal trials in 2026, with significant potential for stock volatility [7] - Intellia Therapeutics (NTLA.US), target price $45: Key data for hereditary angioedema therapy expected in mid-2026, with sufficient cash reserves to support operations [7] - Compass Therapeutics (CMPX.US), target price $8: Data for cholangiocarcinoma therapy expected in early 2026, with a significant market opportunity [8] - KalVista Pharmaceuticals (KALV.US), target price $38: First oral treatment for hereditary angioedema expected to see rapid growth in 2026 [8] Five Potential Acquisition Targets - Jefferies identified five companies that may become acquisition targets in 2026 due to their unique assets or market positions: - Arrowhead Pharmaceuticals (ARWR.US): Strong RNAi product line for cardiovascular and metabolic diseases [9] - Celcuity (CELC.US): Unique efficacy of gedatolisib in breast cancer [9] - ORIC Pharmaceuticals: High-value prostate and lung cancer projects [9] - Travere Therapeutics (TVTX.US): Potential for significant market expansion in rare kidney diseases [9] - KalVista Pharmaceuticals: First-mover advantage in oral HAE treatment [9]