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 condurr脿 attivit脿 di ricerca e sviluppo fino a studi abilitanti per la presentazione dell'IND, dopodich茅 Solid avr脿 opzioni per un ulteriore sviluppo e commercializzazione. La collaborazione utilizzer脿 i capsidi AAV di nuova generazione di Solid, inclusi AAV-SLB101, e capacit脿 di produzione avanzate per affrontare le malattie cardiache di origine genetica. L'azienda prevede di presentare un'IND per il CPVT nella prima met脿 del 2025.
Solid Biosciences (SLDB) ha anunciado una colaboraci贸n estrat茅gica con Mayo Clinic para avanzar en el desarrollo de terapia g茅nica AAV para enfermedades card铆acas. La asociaci贸n otorga a Solid licencias exclusivas a nivel mundial sobre la plataforma de terapia g茅nica Suppression-Replacement de Mayo Clinic y seis programas de terapia g茅nica card铆aca desarrollados por su laboratorio Windland Smith Rice Sudden Death Genomics.
Seg煤n el acuerdo, Mayo Clinic llevar谩 a cabo la investigaci贸n y el desarrollo hasta los estudios que permitan la IND, tras lo cual Solid tendr谩 opciones para continuar con el desarrollo y la comercializaci贸n. La colaboraci贸n utilizar谩 los capsidos AAV de nueva generaci贸n de Solid, incluidos AAV-SLB101, y capacidades de fabricaci贸n avanzadas para abordar enfermedades card铆acas gen茅ticas. La empresa espera presentar una IND para el CPVT en la primera mitad de 2025.
Solid Biosciences (SLDB)电 Mayo Clinic瓿检潣 鞝勲灥鞝 順戨牓鞚 韱淀暣 鞁灔 歆堩櫂鞚 鞙勴暅 AAV 鞙犾爠鞛 旃橂 臧滊皽鞚 於旍頃滊嫟瓿 氚滍憸頄堨姷雼堧嫟. 鞚 韺岉姼雱堨嫮鞚 Solid鞐愱矊 Mayo Clinic鞚 Suppression-Replacement 鞙犾爠鞛 旃橂 頂岆灚韽 氚 Windland Smith Rice Sudden Death Genomics Lab鞐愳劀 臧滊皽頃 6臧滌潣 鞁灔 鞙犾爠鞛 旃橂 頂勲攴鸽灗鞐 雽頃 霃呾爯鞝侅澑 靹戈硠鞝 霛检澊靹检姢毳 攵鞐暕雼堧嫟.
瓿勳暯鞐 霐半澕 Mayo Clinic鞚 IND 鞀轨澑 鞐瓣惮旯岇 鞐瓣惮 氚 臧滊皽鞚 靾橅枆頃橂┌, 鞚错泟 Solid鞚 歆靻嶌爜鞚 臧滊皽 氚 靸侅毄頇 鞓奠厴鞚 臧歆戨媹雼. 鞚 順戨牓鞚 Solid鞚 彀劯雽 AAV 旌§嫓霌, AAV-SLB101鞚 韽暔頃橁碃, 鞙犾爠鞝 鞁灔 歆堩櫂鞚 韮瓴熿溂搿 頃 瓿犼笁 鞝滌“ 電ル牓鞚 頇滌毄頃 瓴冹瀰雼堧嫟. 須岇偓电 2025雲 靸侂皹旮办棎 CPVT鞐 雽頃 IND毳 鞝滌稖頃 鞓堨爼鞛呺媹雼.
Solid Biosciences (SLDB) a annonc茅 une collaboration strat茅gique avec Mayo Clinic pour faire progresser le d茅veloppement de th茅rapies g茅niques AAV pour les maladies cardiaques. Ce partenariat accorde 脿 Solid des licences exclusives dans le monde entier sur la plateforme de th茅rapie g茅nique Suppression-Replacement de la Mayo Clinic et sur six programmes de th茅rapie g茅nique cardiaque d茅velopp茅s par leur laboratoire Windland Smith Rice Sudden Death Genomics.
En vertu de l'accord, le Mayo Clinic m猫nera des recherches et des d茅veloppements jusqu'aux 茅tudes permettant l'IND, apr猫s quoi Solid aura des options pour continuer le d茅veloppement et la commercialisation. La collaboration utilisera les capsides AAV de nouvelle g茅n茅ration de Solid, y compris AAV-SLB101, ainsi que des capacit茅s de fabrication avanc茅es pour cibler les maladies cardiaques d'origine g茅n茅tique. L'entreprise pr茅voit de soumettre un IND pour le CPVT dans la premi猫re moiti茅 de 2025.
Solid Biosciences (SLDB) hat eine strategische Zusammenarbeit mit der Mayo Clinic angek眉ndigt, um die Entwicklung von AAV-Gentherapien f眉r Herzkrankheiten voranzutreiben. Die Partnerschaft gew盲hrt Solid exklusive weltweite Lizenzen f眉r die Suppression-Replacement-Gentherapie-Plattform der Mayo Clinic sowie f眉r 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-erm枚glichenden Studien durchf眉hren, nach denen Solid Optionen f眉r die fortgesetzte Entwicklung und Kommerzialisierung haben wird. Die Zusammenarbeit wird die fortschrittlichen AAV-Capside von Solid, einschlie脽lich AAV-SLB101, sowie moderne Fertigungskapazit盲ten nutzen, um genetische Herzerkrankungen anzugehen. Das Unternehmen plant, in der ersten H盲lfte des Jahres 2025 einen IND f眉r 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鈥檚 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鈥檚 Windland Smith Rice Sudden Death Genomics Lab -
CHARLESTOWN, Mass., Dec. 04, 2024 (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 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, will provide Solid with an exclusive license to their Suppression and Replacement (鈥淪up-Rep鈥) 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鈥檚 鈥淪up-Rep鈥 platform. These programs will use Solid鈥檚 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.
鈥淢ayo Clinic鈥檚 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,鈥 said Michael J. Ackerman, M.D., Ph.D., Genetic Cardiologist and Director of Mayo Clinic鈥檚 Windland Smith Rice Sudden Death Genomics Lab in Rochester, MN. 鈥淚t is gratifying to have the opportunity to work toward developing new therapeutic solutions for patients to help them live and thrive despite their diagnosis.鈥
鈥淕enetic 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,鈥 said Bo Cumbo, President and CEO of Solid. 鈥淏eginning 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.鈥
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鈥檚 mandate is to improve the daily lives of patients living with these devastating 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 our collaborations with Mayo Clinic and other partners, the company鈥檚 goals, priorities and achieve key clinical milestones; the company鈥檚 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鈥檚 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-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 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:
Glenn Silver
FINN Partners
FAQ
What is the purpose of Solid Biosciences' collaboration with Mayo Clinic?
When does SLDB expect to submit its CPVT IND?
What technology licenses did Solid Biosciences obtain from Mayo Clinic?
