Motor neuron connecting to muscle fiber to illustrate myasthenia gravis
Credit: Dr_Microbe/Getty Images

Tevard Biosciences has announced a four-year collaboration with Vertex Pharmaceuticals to develop new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The deal includes options to expand into additional muscular dystrophies and a second indication.

“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.”

The agreement provides Vertex access to Tevard’s proprietary platforms for discovering and developing transfer RNA (tRNA)-based therapies. Tevard will receive up-front, option-exercise, and milestone payments, plus royalties on any approved products. Vertex will fund all program costs and 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. A recent review estimated global prevalence of muscular dystrophies at about 3.6 per 100,000 people. DMD is a severe form of this condition, which primarily affects skeletal muscle. There are many treatments in development for DMD, gene and cell therapies are getting a lot of attention.

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. 

A nonsense mutation is when a sequence of DNA has a stop codon rather than one specifying an amino acid. This error can lead to production of a shortened, non-functional, protein. Tevard aims to use a viral vector to introduce a patented tRNA that will repair this defect. tRNA serves as a link between the messenger RNA (mRNA) molecule and the growing chain of amino acids that make up a protein.

“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, PhD, who chairs the company’s SAB and sits on its board of directors.

Vertex is, ” …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.

Tevard applying its novel Suppressor tRNA, Enhancer tRNA, and mRNA Amplifier platforms in neurological disorders, heart disease, and muscular dystrophies.  

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