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The science of gene therapies is progressing at a remarkable pace, but if these medicines are going to reach patients, trends in investment and commercialization need to progress as well.
The CEOs of three companies involved in genetic medicine discussed the challenges to drug development, investment, scaling manufacturing, and commercialization during a February 26 panel at the BIO CEO & Investor Conference being staged in New York by the Biotechnology Innovation Organization (BIO).
Moderated by Geulah Livshits, Senior Research Analyst of Chardan Capital Markets, the panel included MeiraGTx CEO Alexandra Forbes, LEXEO Therapeutics CEO Nolan Townsend, and Jaguar Gene Therapy CEO Joe Nolan.
Top targets for gene therapy
One consideration for drug development is choosing to target the diseases that are likely to attract investment, make it to the market, and ultimately reach patients. For LEXEO, that generally means lesser-understood diseases for which there is currently no cure, Townsend said. He said these diseases can be more challenging to tackle but also more commercially viable and socially rewarding.
“Diseases that in general do not have existing therapies—it’s a riskier development path, but presumably at the end you have a more commercially attractive disease area,” Townsend said, adding that development for rare diseases also usually has more patient support. “I think in general probably that’s what society wants from our companies is to focus on diseases that have no treatments, and so that’s what should probably be our North Star.”
Nolan said his company’s progression naturally started with rare diseases, but Jaguar Gene Therapy is now looking to move toward diseases with larger patient pools, like heart disease or diabetes.
For Forbes of MeiraGTx, an important consideration is the current state of research and technology for the target disease. “One of the major considerations when we choose an indication is: How optimal is this technology today to address that particular indication?” she said.
Challenges to scaling up manufacturing
Making a gene therapy in the lab is difficult, but producing a sufficient amount for commercialization is also challenging, Livshits noted. She asked if this challenge can be addressed.
“We actually have one of the broadest manufacturing capabilities for AAV verification,” said Forbes, referring to adeno-associated virus (AAV) vectors, the leading platform for gene therapy delivery. She explained that building up the manufacturing side was essential to her company’s progress, as no such manufacturing capability existed. But now that MeiraGTx has developed scalable manufacturing processes with acceptance from regulators, it facilitates further regulatory processes. It also improves the firm’s ability to attract investment, she said.
Jaguar Gene Therapy’s Nolan said that much of the gene therapy development in the recent past has been handled by academics, who did not have to consider the practicalities of production. “But as we get to gene therapies that will be commercialized for much larger patient populations, with pharma companies moving into gene therapy, I think we’re seeing kind of an industrial scale manufacturing,” he said. “And this is happening very quickly.”
Investment, regulation, policy, future outlook
Asked about investor sentiment, panelists said investors appreciate the science, but they need to see faster regulatory processes, and they are also waiting for interest rates to come down. According to Townsend, the more drug companies learn by developing new gene therapies the less the risk, and perceived risk for investors, who become comfortable with the technology.
Panelists said the Food and Drug Administration (FDA) is attempting to build the capacity needed to review new cutting-edge therapies. But thus far, FDA’s capacity has not caught up.
Townsend noted that FDA hold orders, delaying clinical investigations, were much more common for cell and gene therapies because the FDA does not have the knowledge or capacity to review these new fields quickly. He said the FDA just needs to catch up with the technology, and praised the agency’s flexibility in using biomarkers as endpoints for treatments addressing diseases with unmet needs.
Regarding policy changes, panelists said there has been progress in developing appreciation of the true value of genetic therapies, which may have a higher up-front cost but can actually save money in the long run because the one-off therapies can eliminate a disease, meaning no further therapy is needed. According to Nolan, there also needs to be improvements in policy that allows patients and payers to spread out payments over time.
As for the future of gene therapies, Forbes said they are now mostly used to target genetic conditions, but some day they could target as many conditions as small molecule drugs. “What I see in five to 10 years time is using DNA as a therapeutic, maybe much more broadly, to deliver any of the many different proteins or peptides in the body in a way that’s effective and safe,” she said.
