Solid Biosciences Receives FDA Fast Track Designation for SGT-501 First-in-Class Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Solid Biosciences (NASDAQ:SLDB) has received FDA Fast Track designation for SGT-501, its novel gene therapy treatment for catecholaminergic polymorphic ventricular tachycardia (CPVT). This designation adds to previously received FDA IND clearance, Orphan Drug, and Rare Pediatric Disease designations.
SGT-501 is designed as a first-in-class AAV-based gene therapy that delivers a functional CASQ2 gene to cardiac muscle cells, targeting the underlying causes of CPVT, a life-threatening cardiac condition. The therapy has received both FDA IND clearance and Health Canada CTA approval.
The company plans to initiate a Phase 1b clinical trial in Q4 2025, which will be a first-in-human, open-label, multicenter study evaluating the safety, tolerability, and efficacy of SGT-501.
Solid Biosciences (NASDAQ:SLDB) ha ottenuto la designazione FDA Fast Track per SGT-501, il suo innovativo trattamento di terapia genica per la tachicardia ventricolare polimorfa catecolaminergica (CPVT). Questa designazione si aggiunge alle precedenti approvazioni FDA IND, Orphan Drug e Rare Pediatric Disease.
SGT-501 è progettato come una terapia genica AAV di prima classe che introduce un gene CASQ2 funzionale nelle cellule del muscolo cardiaco, agendo sulle cause alla base della CPVT, una condizione cardiaca potenzialmente letale. La terapia ha ricevuto sia l'autorizzazione FDA IND che l'approvazione Health Canada CTA.
L'azienda prevede di avviare uno studio clinico di Fase 1b nel quarto trimestre 2025, uno studio multicentrico, open-label e di prima somministrazione nell'uomo, volto a valutare sicurezza, tollerabilità ed efficacia di SGT-501.
Solid Biosciences (NASDAQ:SLDB) ha recibido la designación FDA Fast Track para SGT-501, su novedoso tratamiento de terapia génica para taquicardia ventricular polimórfica catecolaminérgica (CPVT). Esta designación se suma a las aprobaciones previas de FDA IND, Orphan Drug y Rare Pediatric Disease.
SGT-501 está diseñado como una terapia génica basada en AAV de primera clase que entrega un gen CASQ2 funcional a las células del músculo cardíaco, abordando las causas subyacentes de la CPVT, una condición cardíaca potencialmente mortal. La terapia ha recibido tanto la aprobación FDA IND como la autorización Health Canada CTA.
La compañía planea iniciar un ensayo clínico de Fase 1b en el cuarto trimestre de 2025, un estudio multicéntrico, abierto y de primera administración en humanos para evaluar la seguridad, tolerabilidad y eficacia de SGT-501.
Solid Biosciences (NASDAQ:SLDB)는 카테콜아민성 다형성 심실 빈맥(CPVT) 치료를 위한 새로운 유전자 치료제 SGT-501에 대해 FDA 패스트 트랙 지정을 받았습니다. 이 지정은 이전에 받은 FDA IND 승인, 희귀의약품 및 희귀 소아 질환 지정에 추가됩니다.
SGT-501은 심장 근육 세포에 기능성 CASQ2 유전자를 전달하는 최초의 AAV 기반 유전자 치료제로, 생명을 위협하는 심장 질환인 CPVT의 근본 원인을 겨냥합니다. 이 치료제는 FDA IND 승인과 캐나다 보건청 CTA 승인을 모두 받았습니다.
회사는 2025년 4분기에 1b상 임상시험을 시작할 계획이며, 이는 사람을 대상으로 한 최초의 다기관, 공개 라벨 연구로 SGT-501의 안전성, 내약성 및 효능을 평가할 예정입니다.
Solid Biosciences (NASDAQ:SLDB) a obtenu la désignation FDA Fast Track pour SGT-501, son nouveau traitement de thérapie génique pour la tachycardie ventriculaire polymorphe catécholaminergique (CPVT). Cette désignation s’ajoute aux autorisations précédemment obtenues de la FDA IND, Orphan Drug et Maladie pédiatrique rare.
SGT-501 est conçu comme une thérapie génique de première classe basée sur l’AAV qui délivre un gène CASQ2 fonctionnel aux cellules musculaires cardiaques, ciblant les causes sous-jacentes de la CPVT, une maladie cardiaque potentiellement mortelle. Ce traitement a reçu l’autorisation FDA IND ainsi que l’approbation Health Canada CTA.
L’entreprise prévoit de lancer un essai clinique de phase 1b au quatrième trimestre 2025, une étude multicentrique, ouverte, en première administration chez l’humain, évaluant la sécurité, la tolérance et l’efficacité de SGT-501.
Solid Biosciences (NASDAQ:SLDB) hat für SGT-501, seine neuartige Gentherapie zur Behandlung der katecholaminergen polymorphen ventrikulären Tachykardie (CPVT), die FDA Fast Track-Zulassung erhalten. Diese Zulassung ergänzt die zuvor erhaltenen FDA IND-Freigaben sowie die Orphan Drug- und Rare Pediatric Disease-Designationen.
SGT-501 ist als erstklassige AAV-basierte Gentherapie konzipiert, die ein funktionierendes CASQ2-Gen an Herzmuskelzellen liefert und die zugrundeliegenden Ursachen von CPVT, einer lebensbedrohlichen Herzerkrankung, adressiert. Die Therapie hat sowohl die FDA IND-Freigabe als auch die Health Canada CTA-Zulassung erhalten.
Das Unternehmen plant, im vierten Quartal 2025 eine Phase-1b-Studie zu starten, eine erstmalige Anwendung am Menschen, offene, multizentrische Studie zur Bewertung von Sicherheit, Verträglichkeit und Wirksamkeit von SGT-501.
- None.
- Phase 1b clinical trial yet to begin - early stage of development
- No FDA-approved therapies exist for CPVT, indicating potential regulatory challenges
Insights
Solid Biosciences received FDA Fast Track designation for SGT-501 gene therapy for CPVT, expediting its development path and validating its potential.
The FDA's Fast Track designation for Solid Biosciences' SGT-501 represents a significant regulatory milestone that will expedite the development and review process for this novel gene therapy targeting CPVT. This designation, coupled with previously secured Orphan Drug and Rare Pediatric Disease designations, creates a triple regulatory advantage that enhances the therapy's commercial potential and development pathway.
What makes this development particularly noteworthy is that SGT-501 addresses CPVT - a life-threatening cardiac condition with no FDA-approved therapies currently available. The gene therapy's mechanism of delivering a functional CASQ2 gene to cardiac muscle cells targets the underlying calcium dysregulation that causes dangerous arrhythmias in CPVT patients.
Fast Track benefits include more frequent FDA interactions and potential eligibility for priority review, which could substantially accelerate time-to-market. This marks Solid's third IND clearance in two years, demonstrating consistent regulatory execution capabilities. With both FDA IND clearance and Health Canada CTA approval already secured, the company is positioned to initiate its Phase 1b trial in Q4 2025 as planned. The regulatory momentum builds a strong foundation for the upcoming clinical development phase for this potential first-in-class therapy.
The Fast Track designation for SGT-501 represents a potential breakthrough for CPVT patients who currently lack FDA-approved treatment options. CPVT is characterized by life-threatening arrhythmias triggered by physical or emotional stress, with mortality rates of up to 30-50% in untreated patients.
SGT-501's approach of delivering a functional CASQ2 gene addresses the fundamental pathophysiology of the disease rather than merely managing symptoms. The therapy targets calcium dysregulation and ryanodine receptor instability - the core mechanisms driving arrhythmias in CPVT. Current management relies primarily on beta-blockers, which are often inadequate for complete symptom control and require lifelong adherence.
A successful gene therapy could potentially provide durable treatment with a single administration, transforming the standard of care for these patients. While beta-blockers and implantable defibrillators help manage symptoms and prevent sudden cardiac death, they don't address the underlying genetic cause of calcium mishandling in cardiac cells.
The upcoming Phase 1b trial will be critical to evaluate whether the preclinical promise translates to human subjects. Key efficacy endpoints will likely include reduction in exercise-induced arrhythmias and improvement in cardiac electrical stability. Safety monitoring will be essential, particularly for potential immune responses to the AAV vector and any off-target effects of gene delivery, but the preclinical package appears robust enough to have satisfied regulatory requirements for clinical testing.
- SGT-501 has now received FDA Fast Track, Orphan Drug and Rare Pediatric Disease designations -
- Potential first-in-class therapy with FDA IND clearance and Health Canada CTA approval designed to address underlying causes of CPVT, a life-threatening, arrhythmogenic cardiac condition -
- Solid expects to initiate Phase 1b clinical trial of SGT-501 in the fourth quarter of 2025 -
CHARLESTOWN, Mass., July 23, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a clinical stage life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-501, the Company’s novel, AAV-based investigational gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT). SGT-501 will deliver a functional, full-length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene to cardiac muscle cells, an approach intended to address the underlying ryanodine receptor (RYR2) instability and calcium dysregulation seen in CPVT, thereby normalizing cardiac rhythm in diastole.
Fast Track designation is granted to products that are developed to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. This designation is intended to facilitate development and expedite review of qualifying drugs. SGT-501 will benefit from this designation through more frequent interactions with the FDA along with the potential to be eligible for priority review.
Jessie Hanrahan, Ph.D., Chief Regulatory & Preclinical Operations Officer of Solid Biosciences, commented: “FDA IND clearance and Fast Track designation provide important scientific validation of SGT-501 and recognition of the continuing and severe unmet needs posed by CPVT. We believe SGT-501’s robust preclinical and CMC data package supports the potential for a durable and transformational treatment for a disease that has no FDA-approved therapies. SGT-501 is our third IND clearance in the past two years, demonstrating Solid’s deep regulatory expertise in the genetic medicines space. We want to thank the FDA for their support and partnership and look forward to continued collaboration with regulators globally as we advance our cardiac and neuromuscular candidates.”
FDA Investigational New Drug (IND) clearance and Health Canada Clinical Trial Application (CTA) approval for SGT-501 were announced on July 8, 2025. The planned Phase 1b trial will be a first-in-human, open-label, multicenter study to evaluate the safety, tolerability and efficacy of SGT-501 and is expected to initiate in the fourth quarter of 2025.
About Fast Track Designation
The FDA’s Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.
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 Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA and has potential as a first-in-class therapy to correct the underlying RYR2 instability and calcium dysregulation causes of CPVT.
The AAV-CASQ2 gene therapy approach utilized by SGT-501 was pioneered by Dr. Silvia Priori and her team in IRCCS Maugeri Pavia, Italy. As a globally recognized leader in inherited arrhythmias, Dr. Priori has long been at the forefront of translational cardiac research. Solid Biosciences in-licensed this innovative therapeutic in 2023 and subsequently advanced it into IND-enabling preclinical development, marking an important step toward bringing this potential treatment to individuals living with CPVT.
About Catecholaminergic Polymorphic Ventricular Tachycardia
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 Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, BAG3-mediated dilated cardiomyopathy, and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com.
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 CPVT, 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 Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.
Solid Biosciences Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
investors@solidbio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
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