RARE Daily

Tevard Biosciences Enters Collaboration with Vertex to Develop tRNA-based Therapies for DMD

February 28, 2023

Rare Daily Staff

Tevard Biosciences entered a four-year global research collaboration with Vertex Pharmaceuticals Iaimed at creating new tRNA-based therapies for patients with Duchenne muscular dystrophy caused by nonsense mutations with options to expand into additional muscular dystrophies and a second indication.

The agreement provides access to Tevard’s proprietary platforms for discovering and developing tRNA-based therapies. Under its terms, Tevard will receive upfront, option exercise, and milestone payments, plus royalties on any approved products.

Tevard will advance the research and discovery of novel tRNA-based therapies, with all program costs funded by Vertex. Vertex will be responsible for all subsequent development, manufacturing, and commercialization.

Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy in children, primarily affecting boys, and is caused by a mutation in the dystrophin gene. Patients with DMD are unable to produce normal amounts of dystrophin protein, resulting in progressive muscle weakness and degeneration. Tevard’s tRNA-based therapies may be able to restore dystrophin production in patients whose disease is caused by a nonsense mutation in the dystrophin gene.

Tevard is pioneering tRNA-based therapies to cure a broad range of genetic diseases, exploring the use of its novel Suppressor tRNA, Enhancer tRNA, and mRNA Amplifier platforms in neurological disorders, heart disease, and muscular dystrophies.

“Tevard is devoted to improving the lives of people with severe genetic diseases like Duchenne,” said Daniel Fischer, co-founder and CEO of Tevard. “By combining Tevard’s ability to restore the production of critical proteins with Vertex’s clinical, regulatory, and manufacturing expertise, we hope to make an important difference for patients and their families.”

“Our tRNA-based approach is one of the only methods available to restore production of the full-length dystrophin protein in DMD patients with nonsense mutations. We hope the therapy will be transformative for these patients,” said Tevard co-founder Harvey Lodish, who chairs the company’s SAB and sits on its board of directors.


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