The month of August saw developments across the spectrum of the cell and gene therapy industry — including business, science and regulatory advances.
In early August bluebird bio announced it was partnering with Regeneron to jointly develop new CAR-T therapies, and the pair has already selected the first six targets. The collaboration will draw upon Regeneron’s advanced animal models, allowing for the discovery and characterization of fully human antibodies, as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides.
Elsewhere in business, Sarepta Therapeutics, whose drug eteplirsen was approved by the FDA for DMD in 2016, announced that it was partnering with Lacerta Therapeutics to develop gene therapy treatments for up to three targets within the central nervous system. Lacerta is an AAV-based gene therapy company founded in 2017 with technologies licensed from the University of Florida.
Boston-based Akouos announced in August that it had raised $50 million to develop gene therapies to correct inherited hearing loss. The company hasn’t disclosed its specific disease targets, according to Ryan Cross at C&EN, but has said it will be using synthetic AAVs created in the lab of Luk H. Vandenberghe at Massachusetts Eye & Ear.
In regulatory news, NIH and FDA chiefs announced an important change in the review and regulation of gene therapies. Submissions will no longer go through a special NIH oversight panel, NIH Director Dr. Francis Collins and FDA Chief Dr. Scott Gottlieb wrote in the New England Journal of Medicine. Instead they will go through the same FDA review process as other therapeutics do. The FDA now has more than 700 active INDs for gene therapies (which it classifies to include cell therapies such as CAR-T), and “it seems reasonable to envision a day when gene therapy will be a mainstay of treatment for many diseases,” Collins and Gottlieb wrote.
Finally, a new study has examined the comparative benefits of two types of co-stimulatory domains (elements added to CAR-T therapies to increase their effectiveness). Novartis’ Kymriah uses a 4-1BB co-stimulatory domain, while Gilead’s Yescarta uses CD28. In vitro experiments found that CD28 CAR-T cells attacked cancer cells more quickly and intensely, but in animal models 4-1BB CAR-T cleared cancer cells more effectively, STAT’s Sharon Begley reports. The new study’s findings should be useful “going forward to design safer and more effective” CAR-Ts, said senior author Dr. Stanley Riddell of the Fred Hutchinson Cancer Research Center.