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Gene Therapy Delivery Tech Licensed in $1.8B Deal

DNA puzzle

(Arek Socha, Pixabay)

14 Oct. 2020. A biotechnology company creating synthetic viruses with artificial intelligence to deliver gene therapies is licensing its technology to drug maker Roche. The licensing and collaboration agreement with the Roche Group for diseases affecting the central nervous system and liver is expected to bring Dyno Therapeutics in Cambridge, Massachusetts at least $1.8 billion if all aspects of the deal are fulfilled.

Dyno Therapeutics develops delivery mechanisms for gene therapies, where healthy genes replace inherited mutations responsible for disease. Many current gene therapies use adeno-associated virusesbenign and naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, other than at most mild reactions. In their natural state, however, adeno-associated viruses, or AAVs, an imperfect and inefficient delivery vehicles, on which the company seeks to improve.

The two year-old company is spun-off from the Harvard Medical School genetics lab of George Church that applied machine learning to systematically understand the structure of a key part of AAVs, their outer shell called the capsid. A study by Church and colleagues, published last year in the journal Science, found 735 amino acids making up the capsid’s proteins. Eric Kelsic, a postdoctoral researcher in Church’s lab at the time, led the team, which genetically sequenced and uniquely identified some 200,000 variations of those protein components, and  uncovered a previously unknown protein that helps bind AAVs to their target cells.

Kelsic, Church, and others later founded Dyno Therapeutics, where Kelsic is now CEO. The company extends the academic lab’s work in a technology called CapsidMap that uses machine learning algorithms to design optimized AAV capsids. The algorithms find millions of optimal combinations of targeting ability, payload size, immune evasion, and manufacturing capability, then give each variation a unique DNA identifier. The optimized capsids are then assembled to meet specific therapeutic needs, with each design adding to and refining the algorithms’ experience.

The agreement calls for Dyno Therapeutics to design new AAV capsids that improve gene therapy delivery for treatments affecting the central nervous system, or CNS, and liver. Roche and its subsidiary Spark Therapeutics are responsible for preclinical studies, clinical trials, and commercialization of therapies employing those capsids. Spark Therapeutics is a pioneer in gene therapies acquired last year by Roche that uses AAVs to deliver treatments for rare inherited diseases.

Dyno Therapeutics says the total potential value to the company is at least $1.8 billion, from initial and milestone payments for achieving certain research and clinical objectives, as well as royalties on sales of products from the collaboration. Further details about those payments were not disclosed.

Roche plans to apply Dyno’s technology to its ongoing gene therapy work. “Dyno’s innovative AI-powered approach to designing optimized AAV vectors will further complement and build on our progress in gene therapy,” says James Sabry, head of pharma partnering at Roche in a Dyno Therapeutics statement. “We look forward to leveraging Dyno’s technology to develop new, innovative treatments for patients across CNS and liver-directed therapies.”

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