Emerging Gene Therapy Start-Ups

Lexeo Therapeutics is entering the market with $85 million, 18 gene therapy programs spanning from rare and more prevalent monogenic diseases, and an impressive team. 

Ronald Crystal, a gene therapy veteran founded Lexeo Therapeutic with the National Institutes of Health small business grants in 2018 and is now coming out of stealth with an $85 million in A series cash, 18 preclinical and clinical program that were primarily developed at Weill Cornell Medicine where Crystal is a professor and chairperson of the department of genetic medicine, and the company yields an impressive team that should help to move it forward, recruiting Steven Altschuler, M.D as chairman, and Jay Barth, M.D as chief medical officer. 

“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 the biotech’s lead programs are focussed on the rare disease Friedreich’s ataxia and Batten disease, while it’s 18 programs span rare and more prevalent monogenic disease as well as acquired disease. 

After this round of funding the company plans to push 3 lead programs through the clinic; the Friedreich’s ataxia program is scheduled to enter phase 1 in 2021, and the Batten disease program has just finished a phase ½ study and is set to begin trial in 2022. The third program is being developed in phase 1 with hopes to treat Alzheimer’s disease in those with the APOE4 gene variant which is known to increase the risk for developing the disease. 

The biotech is also going to be utilizing a meaningful portion of the funding to invest in manufacturing, and with its headquarters in New York City, the company has hopes to play a role in building the city’s life science network. 

This is not the only new gene therapy player moving up to the plate, Taysha Gene Therapies launched with $30 million seed financing and 15 programs licensed from UT Southwestern Medical Center, and Kriya Therapeutic debuted with $80.5 million in financing and a mission to develop and advance gene therapies for more complex and highly prevalent serious diseases. 

“Gene therapies have proven to deliver transformational benefit to patients who suffer from devastating diseases with significant, unmet medical need. Our mission at Taysha is to build upon these advancements to eradicate monogenic CNS diseases for the thousands affected,” said RA Session II, President, CEO and Founder of Taysha. “In joining forces with UT Southwestern—home to some of the brightest minds in gene therapy—we will advance our deep pipeline of potentially curative medicines with speed and scale. At Taysha, we are ushering in a new era of gene therapy drug development, one in which we can rapidly translate early discoveries into the clinic and beyond.”

"There have been numerous successful gene therapies focused on rare monogenic diseases in recent years," said Shankar Ramaswamy, M.D., Co-Founder, Chairman, and CEO of Kriya Therapeutics. "We see tremendous potential to expand the field and apply gene therapy to highly prevalent serious diseases. We are focused on designing gene therapies using algorithmic tools, scalable infrastructure, and proprietary technology to optimize the efficacy and durability of our treatments. We look forward to accelerating the development of our pipeline, platform technologies, and internal GMP manufacturing capability with the funds raised in this Series A financing."