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Pharmas License A.I.-Aided Gene Therapies

Retinal gene therapy

(National Human Genome Research Institute, NIH)

11 May 2020. A university spin-off company designing synthetic viruses with artificial intelligence to deliver gene therapies is licensing its work for eye and muscle disorders. Dyno Therapeutics Inc. in Cambridge, Massachusetts, commercializing research from the lab of Harvard University geneticist George Church, says its deals with drug makers Novartis and Sarepta Therapeutics could bring the company as much as $2 billion if all aspects of the agreements 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 viruess, benign 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.

Research by Church and colleagues, reported by Science & Enterprise in November 2019, revealed techniques using machine learning to systematically understand the structure of a key part of AAVs, their outer shell called the capsid. The study, published 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 founded Dyno Therapeutics in May 2018, 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. With machine learning, optimized capsids are assembled to meet specific therapeutic needs, with each design adding to and refining the algorithms’ experience.

Dyno says the agreement with Novartis calls for Dyno to discover and design synthetic viruses to deliver gene therapies for eye diseases, with Novartis responsible for subsequent preclinical, clinical, and commercialization work. While no dollar amounts are disclosed, Dyno is receiving an initial payment, research funding, and license fees, and will be eligible for clinical, regulatory, and sales milestone payments, as well as royalties on sales of products developed.

“Many eye diseases are ideally suited to being treated with gene therapies,” says Kelsic in a company statement, “and more opportunities can be opened with new and improved AAV vectors.” Ophthalmology is one of Novartis’s key disease areas, and the drug maker also is active in cell and gene therapies.

Sarepta Therapeutics develops treatments, including gene therapies, for rare inherited neuromuscular diseases such as Duchenne muscular dystrophy and limb-girdle muscular dystrophy, and more common Charcot-Marie-Tooth disease. In the agreement, Dyno Therapeutics will discover and design optimized synthetic capsids for gene therapies to treat muscle diseases, while Sarepta will be responsible for preclinical, clinical, and commercialization activities.

The deal gives Sarepta an option to license the Dyno technology. During the research phase of the agreement, Dyno will be eligible for more than $40 million in an initial payment, option and license fees from Sarepta. And if Sarepta chooses to adapt Dyno’s gene-therapy capsids for muscle diseases, Dyno will be eligible for undisclosed milestone payments and royalties on product sales.

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