Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia
SLDB 01.07.2025
Drug:SGT-212-001 SGT-212
Diseases:Friedreich’s ataxia
Date of Upcoming Event:2025-01-08
Name of Upcoming Event:Conference Call
About Gravity Analytica
Recent News
- 01.21.2025 - Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
- 01.15.2025 - 43rd Annual J.P. Morgan Healthcare Conference
- 01.15.2025 - Solid Biosciences Outlines Key Priorities for Advancing Diversified Neuromuscular and Cardiac Development Pipeline and Establishing Leadership in Precision Genetic Medicines
Recent Filings
- 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,
FA is a highly complex, multisystem disease that presents distinct challenges for drug development, in part because frataxin is a protein that requires: (1) precise expression levels to avoid fatal cardiac toxicities, and (2) on-target tissue localization in the cerebellum to achieve potential neurological clinical benefit. SGT-212 is the only candidate in development using two routes of administration to address the cardiac manifestations of FA while also directly delivering therapy to the dentate nuclei in the cerebellum, the region most affected and implicated in FA-associated neurologic decline.
In the second half of 2025, the Company expects to initiate a first-in-human, open-label, dose-finding Phase 1b clinical trial of SGT-212. The study will enroll non-ambulatory and ambulatory adult patients living with FA across up to three cohorts and will evaluate the safety and tolerability of contemporaneous systemic and bilateral IDN administration of SGT-212. Participants in the trial will be followed out to five years after receiving SGT-212.
Conference CallThe Company will host a conference call tomorrow,
Participants may also access the live call by dialing 877-409-2291 (toll-free) or 201-389-0925 (international).
About SGT-212SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia (FA) designed to deliver full-length human frataxin (Fxn) via a dual route of administration: intradentate nucleus (IDN) infusion, using an MRI-guided device, followed by an intravenous (IV) infusion to increase therapeutic Fxn levels in the cerebellar dentate nuclei and in the cardiomyocytes, respectively. Restoration of Fxn levels is expected to repair the underlying mitochondrial dysfunction in neurons and cardiomyocytes to address both neurologic and cardiac manifestations of the disease.
SGT-212 was developed by
About Friedreich’s Ataxia (FA)FA is an inherited, life-threatening, degenerative multisystem disease caused by defects in the frataxin gene that disrupt production of the frataxin protein, a mitochondrial iron-binding protein involved in essential cellular processes, including energy production. FA is known to cause progressive nervous system damage, movement problems, and cardiac dysfunction, with cardiac complications identified as the primary cause of death. FA impacts approximately 5,000 people in
About FARAThe Friedreich’s
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Forward-Looking StatementsThis press 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 goals, priorities and achieve key clinical milestones; the Company’s pipeline of programs for neuromuscular and cardiac diseases, including its SGT-212 and SGT-003 programs and expectations for CTA filings, site activations, clinical development, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; the sufficiency of the Company’s cash, cash equivalents, and available-for-sale securities to fund its operations; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s ability to advance SGT-212, SGT-003, SGT-501, SGT-601, SGT-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne, Friedreich’s ataxia and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-212, SGT-003, SGT-501, SGT-601, SGT-401 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the
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