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Dyno advances AI-powered gene therapy

Dyno Therapeutics stepped out of the shadows in May, announcing partnerships with Novartis and Sarepta to use its AI-driven platform to design adeno-associated virus (AAV) vectors for use in gene therapies. The deal with Novartis is centered on eye diseases and the collaboration with Sarepta focuses on muscle disease. For Dyno, which was founded in 2018 with a $9 million venture round from Polaris and CRV, the deals could be worth over $2 billion.

The company is one of 16 startups cofounded in 2018 by George Church, who is based at the Wyss Institute and Harvard Medical School. Dyno drew attention late in 2019 with a publication in Science that demonstrated a machine-guided approach to engineering AAV vector capsids with improved tissue tropism and manufacturability. Several biotechs — including 4DMT, Adverum Biotechnologies, Taysha Gene Therapies and Asklepios BioPharmaceutical — screen large libraries to identify capsids with improved qualities over naturally occurring ones for delivering gene therapies. But Church’s group, which includes Dyno CEO Eric Kelsic and cofounder Sam Sinai, systematically engineered the AAV genome with single codon substitutions, insertions and deletions in the AAV2 capsid followed by high-throughput testing in cells and mice to select the best resulting capsid variants. Machine learning analyzed the capsid libraries optimized for different properties, such as the ability to target the liver or the central nervous system.

In the Novartis collaboration, Dyno will use its platform to develop novel capsids. If drug candidates are developed, Novartis will conduct preclinical and clinical testing of any resulting drug candidates.

Under the Sarepta deal, Dyno will be similarly responsible for generating capsids with improved targeting capabilities in muscles.

In March, Affinia Therapeutics raised $60 million in series A financing to advance its AAV platform, and in April, the company forged a deal with Vertex to engineer new AAV capsids to treat Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.

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Dyno advances AI-powered gene therapy. Nat Biotechnol 38, 661 (2020).

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