Commenting on the path to approval, FDA’s Dr. Marks emphasized the need for a standardized clinical protocol and a framework to streamline the development of more gene therapies.
He also spoke to the recent impact COVID-19 has had on gene therapy reviews, reinforcing the agency’s commitment to move forward submissions as fast as possible despite hiring challenges in the current environment.
The importance of large-cap companies
Dr. Hwang noted the importance of large-cap companies, such as Novartis, Roche, Astellas, Pfizer, etc., which have been investing in independent, public gene therapy companies, such as Spark, Audentes, and Avexis in the last few years.
Holles agreed, saying an acquisition from Astellas was like “rocket fuel” for the company.
How gene therapy can serve patients’ needs
While many patients remain hopeful about gene therapy, Debra Miller offered a compelling patient perspective. She wants the industry to consider those who may not qualify for gene therapy clinical trials or treatments due to their age or possessing existing antibodies.
Other panelists agreed we must look beyond gene therapy and the typical adeno-associated virus (AAV) vector delivery methods. What other methods exist, or can we create, to properly serve patient needs?
Overcome manufacturing issues early
When considering delivery methods, companies must also consider manufacturing capabilities and the need to develop manufacturing experience early on in the process. As Miller noted, delivery is important especially in Duchenne Muscular Dystrophy as corrected genes need to be delivered to muscles all over the body, so this requires a lot of vectors to be produced.
Overcoming manufacturing issues early is critical in establishing safety and effectiveness, both Dr. Marks and Dr. High confirmed. And most importantly, clinical success fuels better investment.
What can the gene therapy industry learn right now?
Holles said the gene therapy industry should see the lack of diversity as an opportunity to bridge the opportunity gap and broaden the type of people recruited within the biotechnology and gene therapy industry.
“This industry has always thrived on doing the right thing and I think we’ve got a real opportunity imposed upon us.”
– Rose Ramseth