Tevard Biosciences and Vertex to develop novel tRNA-based therapies for Duchenne muscular dystrophy

Tevard Biosciences has entered a four-year global research collaboration with Vertex Pharmaceuticals aimed at creating new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) 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 up-front, 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.

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 devoted to improving the lives of people with severe genetic diseases like Duchenne. Given Vertex’s proven track record of developing novel therapies to treat serious disease, we’re thrilled to partner with them on this important effort,” said Tevard CEO and co-founder Daniel Fischer. “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, Ph.D., who chairs the company’s SAB and sits on its board of directors.

“Vertex is committed to transforming the treatment of DMD and we are pursuing various approaches across multiple therapeutic modalities to bring treatments to as many patients as possible. We are excited to partner with Tevard and add their suppressor tRNA technology to our portfolio as another tool we can use to develop treatments for DMD patients with nonsense mutations,” said Mike Cooke, SVP, Vertex Cell and Genetic Therapies.

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