Solid Biosciences Announces FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
- SGT-501 is a novel gene therapy for rare, life-threatening, genetic arrhythmogenic disease with no approved therapies -
- SGT-501 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA -
- Solid expects to initiate Phase 1b clinical trial of SGT-501 in the fourth quarter of 2025 -
- Expands Solid’s clinical pipeline to include first cardiac indication with urgent unmet medical need -
About SGT-501
SGT-501 is an AAV-based gene therapy candidate designed to deliver a functional, full length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene to heart muscle cells. In the context of RYR2 variants, increasing CASQ2 protein levels enhances buffering of free calcium in the sarcoplasmic reticulum, stabilizing the RYR2, which results in reduced diastolic calcium leak into the cytosol. Stabilization of the RYR2 in its closed conformation supports the maintenance of normal cardiac rhythm with the potential to protect against ventricular tachycardia. SGT-501 received Orphan Drug Designation and Rare Pediatric Disease Designation from the
The AAV-CASQ2 gene therapy approach utilized by SGT-501 was pioneered by Dr.
About Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a heart condition marked by abnormal rhythms; specifically, an abnormally fast heartbeat (ventricular tachycardia), which can be triggered by adrenergic stimulation such as physical activity or emotional stress. These arrhythmias can cause unexplained fainting, seizures, cardiac arrest and sudden death. CPVT is estimated to affect roughly 1 in 10,000 individuals globally, although it is often misdiagnosed, and is typically identified in children and young adults. It is mainly caused by mutations in the RYR2 and CASQ2 genes, which disrupt calcium regulation in heart muscle cells, impairing proper heart contraction and relaxation. There are currently no treatments that address the underlying mechanisms of CPVT.
To learn more about CPVT and SGT-501, watch a brief CPVT expert and patient video by clicking here.
About Istituti Clinici Scientifici Maugeri Spa SB (ICS Maugeri)
ICS Maugeri S.p.A. Società Benefit is Italy’s leading healthcare provider specializing in rehabilitation and comprehensive care for vulnerable patients. It stands out for its strong focus on scientific research, operating 18
ICS Maugeri is involved not only in scientific research but also in the application of advanced technologies across medical disciplines, offering personalized care pathways and centers of excellence for specific conditions and clinical specialties. The Group employs more than 4,000 professionals, where medical and professional excellence is intrinsically linked to core values of equity, inclusivity, equality, and transparency - principles that have always guided Maugeri’s mission.
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Forward-Looking Statements
This 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-501, 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-501, SGT-212, SGT-003, 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-501, SGT-212, SGT-003, 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
Solid Biosciences Investor Contact:
Director, Investor Relations and Corporate Communications
investors@solidbio.com
Media Contact:
Yuliya Kutuzava
yuliya.kutuzava@finnpartners.com
Source: Solid Biosciences Inc.