The biotech Sarepta has had a complicated go of it with the FDA sometimes related to their Duchenne muscular dystrophy (DMD) gene therapy efforts. In late 2016 I wrote about how there was some controversy as the FDA approved the Sarepta drug eteplirsen (Exondys 51) also for DMD, going against an advisory panel that had […]
Weekly reads: Sarepta, epigenomics, FDA on eyedrops Read More »
The FDA caused major controversy by approving the drug Exondys 51 from Sarepta Therapeutics for treating Duchenne muscular dystrophy over the recommendations of a scientific panel it convened that had voted 7-3 against approval. Is this contentious Sarepta approval serve as a sign of how the FDA will deal with stem cells moving forward? Will the FDA approval, which
Internal FDA Fighting on Sarepta Bad Omen for Stem Cell Oversight? Read More »
The cell therapy biotech Capricor recently released new data from their Duchenne Muscular Dystrophy (DMD) trial work. The data have generated excitement in the patient advocacy community. The stock has skyrocketed. How exciting are these new data? CEO Linda Marbán is quite upbeat and I think it’s justified. The firm is now seeking FDA approval
Capricor seeks FDA approval of deramiocel for Duchenne based on encouraging data Read More »
Anti-aging might already be the big regenerative medicine story of 2024 and it’s only early March. Anti-aging hype going to the dogs? One of my 20 stem cell and regenerative medicine predictions for 2024 was that longevity would continue to make big news including in some not-so-great ways. In just over two months there has
What are the current FDA Approved Cell and Gene Therapies? In the past many of us have wished there was a list of FDA-approved stem cell therapies. Patients and fellow scientists often asked me, but I couldn’t find a list. As a result, I made a comprehensive list of FDA Approved Cell and Gene Therapies,
List of FDA Approved Cell and Gene Therapies Read More »