Gene therapy veteran Ronald Crystal, M.D., has seen it all.
“I’ve been in the gene therapy field since the beginning, 1987 or so, so I’ve seen the wild period, the dark days, and now we’re back to the wild period,” he said.
And one thing he's realized after being in the field for so long? "In the academic world, we are very, very good at coming up with ideas, developing preclinical evidence and moving to the IND stages. But clinical studies much better belong in a company.”
So, he started one: Lexeo Therapeutics. Founded with National Institutes of Health small business grants in early 2018, the company is coming out of stealth with $85 million in series A cash; 18 preclinical and clinical programs primarily developed at Weill Cornell Medicine, where Crystal is a professor and chairman of the department of genetic medicine; and a star-studded team to move it all forward.
“Our view is the gene therapy field today is very much focused on rare, monogenic diseases,” said Lexeo CEO Nolan Townsend, who previously led rare disease work at Pfizer. “The vision we have for gene therapy is it will eventually move to larger diseases, and we have the pipeline to support that vision.”
Two of Lexeo’s lead programs are in rare diseases: Friedreich’s ataxia and Batten disease. And its 18 programs span rare and more prevalent monogenic diseases—i.e., those caused by a defect in a single gene—as well as acquired diseases, which just means diseases that strike during a person’s lifetime rather than those that affect them from birth.
The funding, drawn from the likes of Longitude Capital, Omega Funds, the Alzheimer’s Drug Discovery Foundation and Alexandria Venture Investments, will push Lexeo’s three lead programs through the clinic. Its Friedreich’s ataxia program is slated to enter phase 1 this year, and its Batten disease treatment has finished a phase 1/2 study and is set for a pivotal trial in 2022. Lexeo is developing its third program for the treatment of Alzheimer’s disease in people with the APOE4 gene, a variant known to increase the risk of developing the disease. It is in phase 1.
Besides bankrolling its clinical trials, Lexeo is using “a meaningful portion” of the funding to invest in manufacturing, a major bottleneck in gene therapy. And that’s not all—the company is headquartered in New York City and hopes to play a role in building the city’s life sciences network.
Joining Crystal and Townsend is chairman Steven Altschuler, M.D., the managing director at Ziff Capital Investments who used to chair the board of gene therapy biotech Spark Therapeutics. The company has also recruited Jay Barth, M.D., as chief medical officer. Barth previously held the same role at Amicus Therapeutics, where he oversaw clinical development in rare disease and gene therapy, including the approval of Fabry disease med Galafold.
Lexeo isn’t the only new gene therapy player on the block. A group of former AveXis executives and investors unveiled their second act in April last year: Taysha Gene Therapies, which launched with $30 million and 15 programs licensed from UT Southwestern Medical Center. The next month, Kriya Therapeutics debuted with $80.5 million and a mission to develop gene therapies for more complex and more common diseases.