CAMBRIDGE, Mass.—Biotechnology company Dyno Therapeutics emerged from stealth mode this week and hit the ground running, announcing two collaborations involving its proprietary technology—one of which is with a Big Pharma partner.
Dyno was founded in 2018 with $9 million from a financing co-led by Polaris Partners and CRV. Its platform, CapsidMap, makes it possible to design novel adeno-associated virus (AAV) vectors that the company says “significantly improve upon current approaches to gene therapy and expand the scope of accessible diseases.”
According to Dyno, naturally occurring AAV vectors are restricted by issues with delivery, immunity, packaging size and manufacturing, which its platform overcomes by using AI technology to design novel capsids, the cell-targeting protein shell of viral vectors. Through DNA library synthesis and next-generation DNA sequencing, in-vivo gene delivery properties are measured in high-throughput. By using AI to comb through data, the CapsidMap technology can generate a map of sequence space to help design and optimize the development of synthetic AAV capsids. This platform builds off of intellectual property from the lab of Dr. George Church, Robert Winthrop Professor of Genetics at Harvard Medical School, a core faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering, and a co-founder of Dyno. The company holds an exclusive option to establish a license agreement with Harvard University for this technology.
“At Dyno, we see a vast opportunity to expand the treatment landscape for gene therapies. The success of gene therapy relies on the ability of vectors to safely and precisely deliver a gene to the intended target cells and tissues,” said Dr. Eric Kelsic, CEO and co-founder of Dyno Therapeutics. “Our approach addresses the major limitations of naturally occurring AAV vectors and creates optimized, disease-specific vectors for gene therapies with great curative potential. Our portfolio of R&D programs and newly announced collaborations with leading gene therapy developers reflect the applicability of our AI-powered approach to improve treatments for patients and expand the number of treatable diseases with gene therapies.”
That portfolio includes programs for ophthalmic, muscle, central nervous system and liver diseases, and as for the new collaborations, those involve Novartis and Sarepta Therapeutics.
The agreement with Novartis is centered on ophthalmology. Per the terms of the deal, Dyno will apply its technology platform to design and discover novel AAV capsids with improved properties for gene therapy. Novaris will assume responsibility for preclinical, clinical and commercialization activities for any resulting product candidates, and will pay Dyno upfront consideration plus committed research funding and license fees. Dyno also stands to receive clinical, regulatory and sales milestone payments, as well as royalties on worldwide net sales of any products commercialized through this agreement.
“We are delighted to be collaborating with Novartis,” Kelsic remarked in a press release. “Many eye diseases are ideally suited to being treated with gene therapies, and more opportunities can be opened with new and improved AAV vectors. With their extensive ophthalmologic expertise, Novartis is an ideal partner to leverage Dyno’s platform to design AI-powered vectors to expand the impact of gene therapies for ocular diseases. This collaboration is a major step forward in our plan to realize the potential of Dyno’s CapsidMap platform for gene therapies to improve patient health.”
As for the deal with Sarepta, that agreement is focused on muscle diseases. Dyno will leverage its CapsidMap technology for the discovery and design of AAV capsids, and similar to the Novartis deal, Sarepta will be responsible for handling preclinical, clinical and commercialization activities. All told, this deal could be worth more than $40 million for Dyno in upfront, option and license payments, and should any products be commercialized, Dyno will be eligible for milestone payments and royalties on worldwide net sales.
“Sarepta’s world-leading gene therapy engine is founded on three pillars: developing a broad portfolio of programs to treat rare diseases; our first-in-class manufacturing expertise; and investment in advancing and further improving the science of gene therapy to help patients in need of more options. To that end, our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases,” commented Doug Ingram, Sarepta’s president and CEO. “By leveraging Dyno’s AI platform and Sarepta’s deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.”
To cap off a busy week, Dyno also presented at the 2020 American Society of Gene and Cell Therapy Conference being held this week. Kelsic made a presentation titled “Artificial intelligence powered design of synthetic AAV capsids without pre-existing immunity for the universal treatment of all patients,” and Dr. Sam Sinai, lead machine learning scientist and co-founder of Dyno, made a second presentation titled “Massively Parallel Deep Diversification of AAV Capsid Proteins by Machine Learning.” Dr. Kathy Lin, computational biology scientist, and Dr. Jeff Gerold, head of Data Science, together presented a poster titled “Accurately Quantifying Transduction within Barcoded AAV Capsid Libraries via Tracking of Single-Molecule ID Tags.”