Ronald Crystal began working in gene therapy in the 1980s, long before the first wave of approvals shook the industry. He took his ideas to Weill Cornell Medicine in 1993, where he helped build a large gene therapy program and spent more than a decade developing potential candidates.

Now, the gene therapy long hauler is launching his own company, Lexeo Therapeutics, with an $85 million Series A to drive three of the companys AAV-administered candidates to market, it said Thursday. Crystal will take the role of chief scientific adviser with Pfizer veteran Nolan Townsend joining as CEO.

These three clinical programs are really the focus of the company and the Series A financing, Townsend told Endpoints News.However, Lexeo has even more candidates waiting in the wings.

Townsend, who hails from Pfizers rare disease program, was introduced to Crystal via a mutual contact. He served in a variety of roles in his 12 years at the pharma giant, including as president of rare disease in North America and other developed markets, country manager in several nations, and director of business operations for Asia-established products. But what attracted him to Lexeo was the opportunity to go after both rare and common diseases.

I saw the potential of this research platform to address a number of rare diseases that do not have adequate therapies today, but also the potential of this platform to address non-rare diseases, he said.

To lead the fledgling team, Crystal and Townsend assembled a seasoned brain trust, including chairman Steven Altschuler, who previously served as chairman of gene therapy pioneer Spark Therapeutics, and PTC Therapeutics vet Jay Barth as executive VP and CMO.

The New York City-based biotech, whose name is a nod to its Lexington Avenue roots, already has two candidates in the clinic: LX1004, which has completed a Phase I/II study and is headed for a pivotal trial in 2022, in CLN2 Batten disease, an autosomal recessive lysosomal storage disease; and LX100, currently in Phase I for APOE4-associated Alzheimers disease. Lexeos third highlighted candidate, an IV-administered treatment for Friedreichs ataxia (FA) dubbed LX2006 is expected to enter Phase I this year. FA is a rare, degenerative multi-system disorder caused by a gene mutation that disrupts the normal production of the protein frataxin, critical to the function of mitochondria in a cell.

The upstart has up to 15 more undisclosed gene therapy programs at different stages of development, according to Townsend and Crystal. Plus, they intend to maintain an ongoing research collaboration with Weill Cornell to bolster their preclinical pipeline.

Lexeos AAV mediated gene therapy programs have the potential for broad applicability across a range of therapeutic indications, and in a single company pipeline present an opportunity for the natural evolution of gene therapy from rare genetic conditions to more common diseases, Crystal said in a statement.

The founder still serves as chairman of Weills Department of Genetic Medicine and will look to continue building Lexeos team in the near future.

We have less than 10 people in the company today. I think were expanding rapidly, so that that number will increase significantly over the next 12 months, Townsend said.

The Series A was led by Longitude Capital and Omega Funds, and joined by Lundbeckfonden Ventures, PBM Capital, Janus Henderson Investors, Invus, Woodline Partners, the Alzheimers Drug Discovery Foundation and Alexandria Venture Investments.

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With decades in gene therapy under his belt, Ronald Crystal launches new venture with up to 18 candidates in the pipe - Endpoints News

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