Subscription Service Details - The Biotech Analysis Central SA marketplace subscription is priced at $49 per month, with a yearly plan offering a 33.50% discount, reducing the cost to $399 per year [1] - A two-week free trial period is currently available for new subscribers [1] Service Offerings - The service provides a deep-dive analysis of numerous pharmaceutical companies [1] - It includes a library of over 600 biotech investing articles, a model portfolio of 10+ small and mid-cap stocks with detailed analysis, live chat, and various analysis and news reports to assist healthcare investors [2] Analyst Background - The author, Terry Chrisomalis, runs the Biotech Analysis Central pharmaceutical service and is the author of the investing group Biotech Analysis Central [1][2]

Core Insights - Solid Biosciences Inc. has received Fast Track designation from the FDA for its gene therapy candidate SGT-212, aimed at treating Friedreich's ataxia (FA) [1][2][3] - SGT-212 is the only therapy in development that targets frataxin deficiency, which is the underlying cause of FA, and aims to address all manifestations of the disease [3][4] - The planned Phase 1b trial will evaluate the safety and tolerability of SGT-212 in adult patients, with dosing expected to start in the second half of 2025 [3][5] Company Overview - Solid Biosciences is focused on developing precision genetic medicines for neuromuscular and cardiac diseases, including FA and Duchenne muscular dystrophy [7][8] - The company aims to improve the lives of patients with rare diseases through innovative gene therapy candidates and technologies [8] Product Details - SGT-212 is a recombinant AAV-based gene replacement therapy designed to deliver the full-length frataxin gene via dual routes: intradentate nucleus infusion and intravenous infusion [5] - The therapy aims to restore frataxin levels to repair mitochondrial dysfunction in neurons and cardiomyocytes, addressing neurological, cardiac, and systemic manifestations of FA [5][6] Disease Context - Friedreich's ataxia is a life-threatening degenerative disease caused by defects in the frataxin gene, affecting approximately 5,000 people in the U.S. and 15,000 in Europe [6] - There are currently no treatments available that can cure or halt the progression of FA [6]

Core Viewpoint - Solid Biosciences Inc. has received Fast Track designation from the FDA for its gene therapy candidate SGT-212, aimed at treating Friedreich's ataxia, marking it as the only dual route gene transfer therapy in development for this condition [1][2][3] Group 1: Product Development - SGT-212 is an AAV-based gene therapy designed to deliver the full-length frataxin gene through intradentate nucleus (IDN) and intravenous (IV) infusions, targeting neurological, cardiac, and systemic manifestations of Friedreich's ataxia [1][5] - The planned Phase 1b trial will be a first-in-human, open-label, multicenter study to evaluate the safety and tolerability of SGT-212, with dosing expected to start in the second half of 2025 [3][4] Group 2: Regulatory Designation - Fast Track designation is granted to products that address serious conditions and unmet medical needs, facilitating expedited development and review processes [2][4] - The designation allows SGT-212 to have more frequent interactions with the FDA and potential eligibility for priority review, which may accelerate its development timeline [2][3] Group 3: Company Background - Solid Biosciences focuses on developing precision genetic medicines for rare neuromuscular and cardiac diseases, including Friedreich's ataxia and Duchenne muscular dystrophy [7][8] - The company aims to improve the lives of patients with devastating rare diseases through innovative gene therapy solutions and advanced delivery platforms [8]

Core Insights - Solid Biosciences Inc. is transitioning into a multi-program leader in precision genetic medicines, focusing on neuromuscular and cardiac diseases, with a corporate update scheduled for the J.P. Morgan Healthcare Conference on January 15, 2025 [1][2] Neuromuscular Pipeline - The company has expanded its pipeline to include gene therapy treatments for Duchenne muscular dystrophy (Duchenne) and Friedreich's ataxia (FA) [3][4] - SGT-003 for Duchenne has dosed four patients in the INSPIRE DUCHENNE clinical trial, showing good tolerance with no serious adverse events (SAEs) reported; initial data from three patients is expected in Q1 2025 [4][5] - SGT-212, the first gene therapy utilizing a dual route of administration for FA, has received FDA IND clearance, with a first-in-human clinical study anticipated in 2H 2025 [4][5] Cardiac Pipeline - SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT) is on track for IND submission in 1H 2025, expanding the clinical-stage portfolio to three unique candidates [4][6] - SGT-601 for TNNT2 Thin Filament Cardiomyopathy is undergoing preclinical IND-enabling studies, with anticipated IND submission in 2H 2026 [6] Financial Position - As of December 31, 2024, the company has approximately $148.9 million in cash and investments, expected to fund strategic priorities into 2026 [4] Collaborations and Innovations - Solid has entered a collaboration with Mayo Clinic to develop an AAV gene therapy platform for genetic cardiomyopathies and channelopathies, receiving an exclusive license for six undisclosed cardiac gene therapy programs [13] - The company is building a library of enabling technologies, including capsids and promoters, with ongoing preclinical studies for immunomodulation [7][13] Manufacturing and Technology - Solid is focused on manufacturing excellence, with improvements in full-to-empty capsid ratios observed in early-stage cardiac programs [8]

CHARLESTOWN, Mass., Jan. 08, 2025 (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 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 3:45 p.m. PT (6:45 p.m. ET). A live webcast of the presentation will be available on the Events page of the Investors section of the Compa ...

Company Update: Solid Biosciences Inc (SLDB) - The FDA has cleared the investigational new drug (IND) application for SGT-212, a novel AAV-based gene therapy candidate targeting Friedreich's ataxia (FA) [1] - SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the cardiac manifestations of FA [1] - The therapy aims to address the neurologic and systemic clinical manifestations of FA, covering the full spectrum of disease progression [2] - Shares of Solid Biosciences surged 17.4% in after-hours trading on Jan 7 and continued to rise in pre-market trading on Jan 8 [2] - Over the past year, SLDB shares have declined 51.4%, significantly underperforming the industry's 15.7% decline [2] Clinical Development of SGT-212 - The IND is supported by robust preclinical data on SGT-212 [3] - A phase Ib study for SGT-212 is planned to initiate in the second half of 2025 [3] - The study will evaluate the safety and tolerability of systemic intravenous infusion and direct intradentate nuclei (IDN) infusion in both non-ambulatory and ambulatory adult FA patients [3] - The dual route of administration enables direct delivery of SGT-212 to the heart and cerebellum, potentially addressing the most significant symptoms of FA [4] - SGT-212 represents a differentiated approach to treating the underlying disease [4] Competitive Landscape in FA Market - FA is a rare disease, with limited companies developing treatments [7] - Biogen (BIIB) markets Skyclarys, the first FDA-approved treatment for FA, approved in 2023 in the US and February 2024 in Europe [7] - Skyclarys was added to Biogen's portfolio through the acquisition of Reata Pharmaceuticals in September 2023 [7] - Biogen reports strong progress in the US and EU launch of Skyclarys, with increasing global demand [8] - Larimar Therapeutics (LRMR) is developing nomlabofusp (CTI-1601) as a potential FA treatment, with plans to file a biologics license application in the second half of 2025 [8] - Nomlabofusp has the potential to be the first frataxin protein replacement therapy for FA patients [9] Stock Performance and Industry Comparison - Solid Biosciences currently holds a Zacks Rank 3 (Hold) [10] - Puma Biotechnology (PBYI), a better-ranked stock in the biotech sector, holds a Zacks Rank 1 (Strong Buy) [10] - PBYI's 2025 earnings per share estimates have increased from 42 cents to 54 cents over the past 60 days [10] - PBYI shares have declined 21.7% over the past year [10] - Puma Biotechnology has beaten earnings estimates in three of the trailing four quarters, with an average surprise of 32.78% [11]

- SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia - - Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease - - Phase 1b clinical trial initiation expected in 2H 2025 - - Company to hold a conference call tomorrow, January 8, 2025, at 8:30 AM ET - CHARLESTOWN, Mass., Jan. 07, 2025 (GLOBE NEWSWI ...

- SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich's ataxia - - Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease - - Phase 1b clinical trial initiation expected in 2H 2025 - - Company to hold a conference call tomorrow, January 8, 2025, at 8:30 AM ET - CHARLESTOWN, Mass., Jan. 07, 2025 (GLOBE NEWSWI ...

CHARLESTOWN, Mass., Dec. 23, 2024 (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 it was added to the Nasdaq Biotechnology Index® (Nasdaq: NBI) effective prior to the market open on Monday, December 23, 2024. The Nasdaq Biotechnology Index is designed to track the performance of a set of securities listed on The Nasdaq Stock Market® (Nasdaq®) that ...

Core Viewpoint - Wedbush initiated coverage on Solid Biosciences, Inc., a gene therapy company focused on treating Duchenne muscular dystrophy (DMD) [1] Company Overview - Solid Biosciences is developing SGT-003, the leading program in its pipeline, with plans to advance additional projects toward clinical trials in 2025 [2] - The therapy utilizes AAV-SLB101 capsid and a specialized cargo (nNOS binding domain) to potentially enhance dystrophin expression compared to earlier treatments [2][3] Market Potential - The analyst notes that while first-generation gene therapies for DMD have opened new treatment possibilities, there is still significant room for improvement [3] - Solid Biosciences' innovative approach and novel capsid technology could improve microdystrophin delivery [3] Milestones and Projections - The initial Phase 1/2 INSPIRE DUCHENNE trial results are expected in the first quarter of 2025, marking a significant near-term milestone for the company [4] - The approval of Sarepta Therapeutics Inc's Elevidys is seen as a key milestone, but the market still requires newer treatments with better results [4] Financial Outlook - Solid Biosciences' stock is currently trading around $5, with approximately $4 per share in cash providing some downside protection [4] - The company ended Q3 2024 with about $171.1 million in cash and equivalents, with a cash runway anticipated into 2026 [6] - Assuming SGT-003 follows a timeline similar to Sarepta's Elevidys, even a one-year delay would lower the price target to $11 per share, still above the current trading price of $4.83 [5][7] Analyst Rating - The analyst sees strong potential in Solid Biosciences and has initiated coverage with an Outperform rating [5]