Solid Biosciences Announces FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Solid Biosciences (NASDAQ:SLDB) has received both FDA IND and Health Canada CTA approvals for SGT-501, their first-in-class cardiac gene therapy designed to treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). The therapy has secured Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.
CPVT is a rare, life-threatening genetic heart condition with no currently approved therapies. The company plans to initiate a Phase 1b clinical trial in Q4 2025 to evaluate SGT-501's safety, tolerability, and efficacy. The therapy targets the underlying disease mechanism by addressing abnormal calcium releases from the sarcoplasmic reticulum in the heart.
Solid Biosciences (NASDAQ:SLDB) ha ottenuto le approvazioni FDA IND e Health Canada CTA per SGT-501, la loro terapia genica cardiaca innovativa progettata per trattare la Tachicardia Ventricolare Polimorfa Catecolaminergica (CPVT). La terapia ha ricevuto la Designazione di Farmaco Orfano e la Designazione di Malattia Pediatrica Rara dalla FDA.
La CPVT 猫 una rara e pericolosa condizione genetica del cuore per cui non esistono terapie approvate. L'azienda prevede di avviare una fase 1b di sperimentazione clinica nel quarto trimestre del 2025 per valutare la sicurezza, la tollerabilit脿 e l鈥檈fficacia di SGT-501. La terapia agisce sul meccanismo alla base della malattia, correggendo le anomalie nel rilascio di calcio dal reticolo sarcoplasmatico nel cuore.
Solid Biosciences (NASDAQ:SLDB) ha recibido las aprobaciones FDA IND y Health Canada CTA para SGT-501, su terapia g茅nica card铆aca innovadora dise帽ada para tratar la Taquicardia Ventricular Polim贸rfica Catecolamin茅rgica (CPVT). La terapia ha obtenido la Designaci贸n de Medicamento Hu茅rfano y Designaci贸n de Enfermedad Pedi谩trica Rara por parte de la FDA.
La CPVT es una enfermedad gen茅tica card铆aca rara y potencialmente mortal, para la cual no existen terapias aprobadas actualmente. La compa帽铆a planea iniciar un ensayo cl铆nico de fase 1b en el cuarto trimestre de 2025 para evaluar la seguridad, tolerabilidad y eficacia de SGT-501. La terapia aborda el mecanismo subyacente de la enfermedad corrigiendo las liberaciones anormales de calcio desde el ret铆culo sarcopl谩smico en el coraz贸n.
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CPVT電� 順勳灛 鞀轨澑霅� 旃橂鞝滉皜 鞐嗠姅 頋穩頃橁碃 靸濍獏鞚� 鞙勴槕頃橂姅 鞙犾爠靹� 鞁灔 歆堩櫂鞛呺媹雼�. 須岇偓電� 2025雲� 4攵勱赴鞐� 1b靸� 鞛勳儊鞁滍棙鞚� 鞁滌瀾頃橃棳 SGT-501鞚� 鞎堨爠靹�, 雮挫暯靹� 氚� 須姤鞚� 韽夑皜頃� 瓿勴殟鞛呺媹雼�. 鞚� 旃橂鞝滊姅 鞁灔 雮� 攴检唽韽泊鞐愳劀 牍勳爼靸侅爜鞚� 旃检姌 氚╈稖鞚� 臁办爤頃橃棳 歆堧硲鞚� 攴茧掣 鞗愳澑鞚� 韮�旯冹溂搿� 頃╇媹雼�.
Solid Biosciences (NASDAQ:SLDB) a obtenu les autorisations FDA IND et Health Canada CTA pour SGT-501, leur th茅rapie g茅nique cardiaque innovante con莽ue pour traiter la tachycardie ventriculaire polymorphe cat茅cholaminergique (CPVT). Cette th茅rapie a re莽u la d茅signation de m茅dicament orphelin et la d茅signation de maladie p茅diatrique rare de la FDA.
La CPVT est une maladie g茅n茅tique cardiaque rare et potentiellement mortelle, sans traitement approuv茅 脿 ce jour. La soci茅t茅 pr茅voit de lancer un essai clinique de phase 1b au quatri猫me trimestre 2025 afin d鈥櫭﹙aluer la s茅curit茅, la tol茅rance et l鈥檈fficacit茅 de SGT-501. Cette th茅rapie cible le m茅canisme sous-jacent de la maladie en corrigeant les lib茅rations anormales de calcium provenant du r茅ticulum sarcoplasmique dans le c艙ur.
Solid Biosciences (NASDAQ:SLDB) hat sowohl die FDA IND- als auch die Health Canada CTA-Zulassungen f眉r SGT-501 erhalten, ihre neuartige kardiale Gentherapie zur Behandlung der katecholaminergen polymorphen ventrikul盲ren Tachykardie (CPVT). Die Therapie erhielt von der FDA die Orphan-Drug- und Rare Pediatric Disease Designation.
CPVT ist eine seltene, lebensbedrohliche genetische Herzerkrankung, f眉r die derzeit keine zugelassenen Therapien existieren. Das Unternehmen plant, im vierten Quartal 2025 eine Phase-1b-Studie zu starten, um Sicherheit, Vertr盲glichkeit und Wirksamkeit von SGT-501 zu bewerten. Die Therapie zielt auf den zugrundeliegenden Krankheitsmechanismus ab, indem sie abnormale Kalziumfreisetzungen aus dem sarkoplasmatischen Retikulum im Herzen adressiert.
- FDA IND and Health Canada CTA approvals received for SGT-501
- Secured both Orphan Drug and Rare Pediatric Disease Designations
- Addresses an unmet medical need with no current FDA-approved treatments
- Expands pipeline into cardiac indications
- Built on proven research demonstrating therapeutic impact in cellular and mouse models
- Phase 1b clinical trials not yet initiated
- Early-stage development with no human efficacy data available yet
Insights
Solid Biosciences achieves key regulatory approvals for CPVT gene therapy, advancing first-in-class treatment for fatal cardiac arrhythmia toward clinical trials.
Solid Biosciences has secured a significant regulatory milestone with both FDA IND and Health Canada CTA approvals for SGT-501, their novel gene therapy targeting Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). This rare cardiac disorder, characterized by potentially fatal arrhythmias triggered by emotional or physical stress, currently has no FDA-approved treatments despite being identified nearly 50 years ago.
The scientific approach behind SGT-501 is particularly innovative, targeting the disease's underlying mechanism: abnormal calcium release from the sarcoplasmic reticulum caused by mutations in the cardiac ryanodine receptor (RYR2). The therapy appears to leverage calsequestrin overexpression, based on foundational research from Dr. Priori's laboratory that demonstrated efficacy in both cellular and animal models.
The regulatory support for this program is substantial, with the FDA granting both Orphan Drug Designation and Rare Pediatric Disease Designation. These designations provide significant incentives including market exclusivity periods and potential priority review vouchers, enhancing the commercial potential if SGT-501 proves successful in clinical development.
The company plans to initiate a Phase 1b trial in Q4 2025, which will evaluate safety, tolerability, and efficacy. This trial represents a critical inflection point, as positive data could position Solid Biosciences as a pioneer in addressing this life-threatening condition. For CPVT patients, who currently rely on beta-blockers and lifestyle modifications with variable success, SGT-501 could potentially offer the first disease-modifying treatment that addresses the genetic root cause rather than merely managing symptoms.
- 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鈥檚 clinical pipeline to include first cardiac indication with urgent unmet medical need -
CHARLESTOWN, Mass., July 08, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the 鈥淐ompany鈥� or 鈥淪olid鈥�), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced approval of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) and clinical trial application (CTA) by Health Canada for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by genetic mutations that impact the ryanodine receptor (RYR2) in cardiac muscle. The Company expects to initiate a Phase 1b clinical trial to evaluate the safety, tolerability and efficacy of SGT-501 in the fourth quarter of 2025.
Gabriel Brooks, M.D., Chief Medical Officer of Solid, said: 鈥淒espite being identified nearly 50 years ago, CPVT still lacks FDA-approved therapies 鈥� this announcement reflects a critical development in the treatment of this underserved, often fatal, cardiac disease. SGT-501 offers a precision genetic approach targeting the underlying pathophysiology of the disease: abnormal calcium releases from the sarcoplasmic reticulum in an otherwise structurally sound heart. We believe SGT-501 has the unique potential to provide durable protection and may be capable of liberating patients from the ever-present threat of lethal arrhythmias and life-limiting prohibitions on exercise. Solid is proud to launch this program to help address this clear unmet need, further expanding our pipeline of differentiated and thoughtfully designed genetic medicines.鈥�
Silvia Priori, M.D., Ph.D., Professor of Cardiology at the University of Pavia and Director of the Molecular Cardiology Unit at the IRCCS Maugeri in Pavia, Italy, said: 鈥淎fter decades during which we, the clinical community, have been limited in our ability to treat people living with CPVT, I have long hoped for the day when a genetic-modifying therapy becomes available. We are proud to partner with Solid who advanced fundamental work from our labs at Maugeri, which demonstrated the principle that calsequestrin overexpression can have a therapeutic impact on multiple forms of CPVT in both cellular and mouse models of disease. This work was further developed into a compelling IND safety and efficacy package through close collaboration with, and studies conducted by, the Solid team. I look forward to clinical updates for SGT-501 and seeing how this potential medicine may benefit people living with CPVT who are in critical need of disease-specific medicines.鈥�
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 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 the IRCCS ICS Maugeri lab in 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 (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
About Istituti Clinici Scientifici Maugeri Spa SB (ICS Maugeri)
ICS Maugeri S.p.A. Societ脿 Benefit is Italy鈥檚 leading healthcare provider specializing in rehabilitation and comprehensive care for vulnerable patients. It stands out for its strong focus on scientific research, operating 18 Clinical Institutes - including 9 recognized as IRCCS (Scientific Institutes for Research, Hospitalization and Healthcare) and 9 as Scientific Clinical Institutes -along with 6 outpatient centers, 1 Environmental Research Center, and a Poison Control Center and National Toxicology Information Center.
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鈥檚 mission.
Headquartered in Pavia, ICS Maugeri operates 23 healthcare facilities across 7 Italian regions, including 9 IRCCS, 10 Scientific Clinical Institutes, 5 outpatient clinics, a Poison Control Center 鈥揟oxicology Center, and an Environmental Research Center.
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鈥檚 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鈥檚 mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit .
Forward-Looking Statements
This press release contains 鈥渇orward-looking statements鈥� 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鈥檚 goals, priorities and achieve key clinical milestones; the Company鈥檚 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鈥檚 cash, cash equivalents, and available-for-sale securities to fund its operations; and other statements containing the words 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渃ontinue,鈥� 鈥渃ould,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥渋ntend,鈥� 鈥渕ay,鈥� 鈥減lan,鈥� 鈥減otential,鈥� 鈥減redict,鈥� 鈥減roject,鈥� 鈥渟hould,鈥� 鈥渢arget,鈥� 鈥渨ould,鈥� 鈥渨orking鈥� and similar expressions. Any forward-looking statements are based on management鈥檚 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鈥檚 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鈥檚 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鈥檚 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鈥檚 actual results to differ from those contained in the forward-looking statements, see the 鈥淩isk Factors鈥� section, as well as discussions of potential risks, uncertainties and other important factors, in the company鈥檚 most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company鈥檚 views as of the date hereof and should not be relied upon as representing the company鈥檚 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 Investor Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
Media Contact:
Yuliya Kutuzava
FINN Partners
FAQ
What approvals did Solid Biosciences (SLDB) receive for SGT-501?
When will Solid Biosciences begin clinical trials for SGT-501?
What is CPVT and how does SGT-501 aim to treat it?
What special designations has SGT-501 received from the FDA?
How is SGT-501 different from current CPVT treatments?
