It’s been a busy month for companies working on cell and gene therapies, as the industry barrels full-steam ahead.
We’ll start with some good news from Australia, where Mesoblast, a company working on an allogeneic stem cell product to treat graft-versus-host disease (GVHD), reported positive results from a phase 3 trial. GVHD, which causes inflammation and tissue damage, often afflicts blood cancer patients after they’ve had hematopoietic cell transplants and has been associated with increased mortality. Mesoblast’s off-the-shelf treatment, called remestemcel-L, stimulated a complete or partial response in 69 percent of children treated in an open-label trial. According to Endpoints News, the company is seeking swift approval from the U.S. Food and Drug Administration.
Next, we’ll explore several financing deals that indicate just how hot the cell and gene therapy sector continues to be.
Gilead Sciences struck a much-talked-about deal to gain access to gene editing technology held by Sangamo Therapeutics. Gilead’s Kite division will use the zinc finger nuclease technology to develop new CAR-T therapies, FierceBiotech reports. Kite is paying Sangamo $150 million upfront, with the promise of up to $3 billion more if the company hits key milestones in developing products. Zinc finger editing preceldates CRISPR/Cas9 and has occasionally been dismissed as old tech, but this deal suggests that it still has value for drug development.
In another interesting deal, reported by Endpoints News, Bioverativ, a gene therapy company working on hemophilia treatments, has agreed to pay Oxford BioMedica up to $100 million to use its lentivirus platform as a vector to get DNA into patients’ cells. Many other gene therapy companies are using adeno-associated viruses as vectors. Bioverativ, however, joins bluebird bio, Novartis and others in deploying lentiviruses.
Also on the financing front: Investors poured $250 million into Celularity, a New Jersey startup building a stem cell platform to develop treatments in the areas of immuno-oncology, regenerative medicine and longevity. The company, a Celgene spinoff, has some 200 patents (either pending or already issued). Its big innovation: using placental cells as the building blocks for therapies. Endpoints News reports that it’s working, for instance, on off-the-shelf CAR-T cancer treatments that are derived from placenta cells, rather than custom-built from the patient’s own T cells.
We’ll end our monthly news roundup by highlighting an encouraging story from STAT that lists a half-dozen gene therapies in the works for sickle cell disease. Companies including CRISPR Therapeutics, Editas Medicine, bluebird bio and Sangamo Therapeutics are all racing to develop treatments, STAT reports. It’s a welcome development, especially given how long sickle cell disease — which can cause crippling pain and organ damage – has been overlooked. We’re looking forward to hearing more as the research progresses.