Solid Biosciences Announces Collaboration with Mayo Clinic to Accelerate Gene Therapy Innovation and Advance Development of Cardiac Gene Therapies
Solid Biosciences (SLDB) has announced a strategic collaboration with Mayo Clinic to advance AAV gene therapy development for cardiac diseases. The partnership grants Solid exclusive worldwide licenses to Mayo Clinic's Suppression-Replacement gene therapy platform and six cardiac gene therapy programs developed by their Windland Smith Rice Sudden Death Genomics Lab.
Under the agreement, Mayo Clinic will conduct research and development up to IND-enabling studies, after which Solid will have options for continued development and commercialization. The collaboration will utilize Solid's next-generation AAV capsids, including AAV-SLB101, and advanced manufacturing capabilities to target genetic heart diseases. The company expects to submit an IND for CPVT in the first half of 2025.
Solid Biosciences (SLDB) ha annunciato una collaborazione strategica con Mayo Clinic per far progredire lo sviluppo della terapia genica AAV per le malattie cardiache. La partnership concede a Solid licenze esclusive a livello mondiale sulla piattaforma di terapia genica Suppression-Replacement della Mayo Clinic e sei programmi di terapia genica cardiaca sviluppati dal loro Windland Smith Rice Sudden Death Genomics Lab.
In base all'accordo, la Mayo Clinic condurrà attività di ricerca e sviluppo fino a studi abilitanti per la presentazione dell'IND, dopodiché Solid avrà opzioni per un ulteriore sviluppo e commercializzazione. La collaborazione utilizzerà i capsidi AAV di nuova generazione di Solid, inclusi AAV-SLB101, e capacità di produzione avanzate per affrontare le malattie cardiache di origine genetica. L'azienda prevede di presentare un'IND per il CPVT nella prima metà del 2025.
Solid Biosciences (SLDB) ha anunciado una colaboración estratégica con Mayo Clinic para avanzar en el desarrollo de terapia génica AAV para enfermedades cardíacas. La asociación otorga a Solid licencias exclusivas a nivel mundial sobre la plataforma de terapia génica Suppression-Replacement de Mayo Clinic y seis programas de terapia génica cardíaca desarrollados por su laboratorio Windland Smith Rice Sudden Death Genomics.
Según el acuerdo, Mayo Clinic llevará a cabo la investigación y el desarrollo hasta los estudios que permitan la IND, tras lo cual Solid tendrá opciones para continuar con el desarrollo y la comercialización. La colaboración utilizará los capsidos AAV de nueva generación de Solid, incluidos AAV-SLB101, y capacidades de fabricación avanzadas para abordar enfermedades cardíacas genéticas. La empresa espera presentar una IND para el CPVT en la primera mitad de 2025.
Solid Biosciences (SLDB)는 Mayo Clinic과의 전략적 협력을 통해 심장 질환을 위한 AAV 유전자 치료 개발을 추진한다고 발표했습니다. 이 파트너십은 Solid에게 Mayo Clinic의 Suppression-Replacement 유전자 치료 플랫폼 및 Windland Smith Rice Sudden Death Genomics Lab에서 개발한 6개의 심장 유전자 치료 프로그램에 대한 독점적인 세계적 라이센스를 부여합니다.
계약에 따라 Mayo Clinic은 IND 승인 연구까지 연구 및 개발을 수행하며, 이후 Solid은 지속적인 개발 및 상용화 옵션을 가집니다. 이 협력은 Solid의 차세대 AAV 캡시드, AAV-SLB101을 포함하고, 유전적 심장 질환을 타겟으로 한 고급 제조 능력을 활용할 것입니다. 회사는 2025년 상반기에 CPVT에 대한 IND를 제출할 예정입니다.
Solid Biosciences (SLDB) a annoncé une collaboration stratégique avec Mayo Clinic pour faire progresser le développement de thérapies géniques AAV pour les maladies cardiaques. Ce partenariat accorde à Solid des licences exclusives dans le monde entier sur la plateforme de thérapie génique Suppression-Replacement de la Mayo Clinic et sur six programmes de thérapie génique cardiaque développés par leur laboratoire Windland Smith Rice Sudden Death Genomics.
En vertu de l'accord, le Mayo Clinic mènera des recherches et des développements jusqu'aux études permettant l'IND, après quoi Solid aura des options pour continuer le développement et la commercialisation. La collaboration utilisera les capsides AAV de nouvelle génération de Solid, y compris AAV-SLB101, ainsi que des capacités de fabrication avancées pour cibler les maladies cardiaques d'origine génétique. L'entreprise prévoit de soumettre un IND pour le CPVT dans la première moitié de 2025.
Solid Biosciences (SLDB) hat eine strategische Zusammenarbeit mit der Mayo Clinic angekündigt, um die Entwicklung von AAV-Gentherapien für Herzkrankheiten voranzutreiben. Die Partnerschaft gewährt Solid exklusive weltweite Lizenzen für die Suppression-Replacement-Gentherapie-Plattform der Mayo Clinic sowie für sechs von deren Windland Smith Rice Sudden Death Genomics Lab entwickelte Programme zur Gentherapie bei Herzerkrankungen.
Im Rahmen der Vereinbarung wird die Mayo Clinic Forschungs- und Entwicklungsarbeiten bis hin zu IND-ermöglichenden Studien durchführen, nach denen Solid Optionen für die fortgesetzte Entwicklung und Kommerzialisierung haben wird. Die Zusammenarbeit wird die fortschrittlichen AAV-Capside von Solid, einschließlich AAV-SLB101, sowie moderne Fertigungskapazitäten nutzen, um genetische Herzerkrankungen anzugehen. Das Unternehmen plant, in der ersten Hälfte des Jahres 2025 einen IND für CPVT einzureichen.
- Secured exclusive worldwide licenses for Mayo Clinic's gene therapy platform
- Obtained rights to develop and commercialize six cardiac gene therapy programs
- Access to Mayo Clinic's research capabilities through IND-enabling studies
- None.
Insights
This collaboration marks a significant strategic expansion in Solid Biosciences' cardiac gene therapy portfolio. The exclusive licensing of Mayo Clinic's Suppression-Replacement platform, along with six cardiac therapy programs, substantially strengthens SLDB's position in treating genetic heart conditions. The partnership leverages SLDB's proprietary AAV capsids, particularly AAV-SLB101, combining it with Mayo's expertise in cardiac genomics.
The near-term catalyst is the planned CPVT IND submission in H1 2025. The collaboration provides multiple shots on goal in treating genetic cardiomyopathies and channelopathies, potentially addressing significant unmet medical needs. Mayo Clinic's involvement adds considerable credibility and expertise to SLDB's cardiac program development.
This strategic partnership could significantly enhance SLDB's market position in the cardiac gene therapy space. For a company with a market cap of
The collaboration diversifies SLDB's pipeline beyond its current focus, potentially creating multiple value-driving catalysts. The addressable market for genetic cardiac conditions is significant, though specific revenue projections would depend on individual program success rates and pricing strategies.
- Solid Biosciences to collaborate with Mayo Clinic’s genetic cardiology disease laboratory -
- Solid to receive exclusive worldwide licenses to AAV-based Suppression-Replacement gene therapy platform and multiple next-generation cardiac gene therapy programs developed by Mayo Clinic’s Windland Smith Rice Sudden Death Genomics Lab -
CHARLESTOWN, Mass., Dec. 04, 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 a collaboration with Mayo Clinic seeking to advance a cutting-edge AAV gene therapy platform for the development of next-generation therapies to treat sudden cardiac death-predisposing genetic cardiomyopathies and channelopathies with high unmet medical need and significant patient populations.
Under the terms of the agreement, Mayo Clinic’s Windland Smith Rice Sudden Death Genomics Laboratory will provide Solid with an exclusive license to their Suppression and Replacement (“Sup-Rep”) gene therapy platform. The collaboration will also provide Solid with an exclusive license to develop and commercialize six cardiac gene therapy programs developed using Mayo Clinic’s “Sup-Rep” platform. These programs will use Solid’s next-generation AAV capsids, including AAV-SLB101, and advanced manufacturing capabilities to suppress and replace genes implicated in potentially life-threatening genetic heart diseases. Mayo Clinic will be responsible for research and development efforts for each cardiac gene therapy candidate up to IND-enabling studies, at which point, Solid will have the option to pursue continued development and commercialization of each licensed program.
“Mayo Clinic’s suppression-replacement gene therapy platform has the potential for a novel way of treating patients at risk for sudden cardiac death from their genetic heart disease,” said Michael J. Ackerman, M.D., Ph.D., Genetic Cardiologist and Director of Mayo Clinic’s Windland Smith Rice Sudden Death Genomics Lab in Rochester, MN. “It is gratifying to have the opportunity to work toward developing new therapeutic solutions for patients to help them live and thrive despite their diagnosis.”
“Genetic cardiomyopathies and channelopathies represent the next frontier for the gene therapy field, and our collaboration with Dr. Ackerman and his research team places Solid at the forefront of innovation in this space,” said Bo Cumbo, President and CEO of Solid. “Beginning with our CPVT IND submission expected in the first half of 2025, followed by the development of the Sup-Rep AAV platform and multiple additional therapies targeting high-impact indications, we have laid the foundation for Solid to become the leader in cardiac precision genetic medicine. We look forward to working alongside Mayo Clinic and Dr. Ackerman to transform cardiac patient care.”
Mayo Clinic has a financial interest in the Company and technology referenced in this press release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.
About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for the treatment of TNNT2-mediated dilated cardiomyopathy, SGT-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating 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 our collaborations with Mayo Clinic and other partners, the company’s goals, priorities and achieve key clinical milestones; the company’s SGT-003 program, including expectations for additional CTA filings, site activations, expanded clinical development, accelerated production of multiple GMP batches of SGT-003, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; the company’s expectations for submission of an IND for SGT-501 and to submit additional INDs by the end of 2026; the cash runway of the company and 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-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 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-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 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 Investor 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
FAQ
What is the purpose of Solid Biosciences' collaboration with Mayo Clinic?
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