September 18, 2020

The Niche

Knoepfler lab stem cell blog

Duchenne Muscular Dystrophy

2 min read

This post includes my updated list of firms/products that have received FDA regenerative medicine advanced therapy designation or RMAT, which are in the public domain. The numbers really jumped in the last month. I’m assuming for the purposes of this resource page that all self-reports are accurate. Note that the links embedded into the firm names are to relevant press releases or other reference material. A special thanks to some folks at ARM for helping me keep the list up to date. You can …Read More

2 min read

How is the stem cell and regenerative medicine biotech sector doing these days and does recent news about specific biotechs such as Capricor suggest issues for the sector more broadly? Capricor had bad news earlier this year related to its stem cells for heart disease program and things just took a turn for the worse this week as pharma giant J&J severed ties with the small California company (here and here). Capricor, which has received more than $17 million in CIRM grant funding, could …Read More

2 min read

Over at the California Stem Cell Report, David Jensen is reporting on some good news from CIRM for California on the stem cell clinical trial front. Stem cell biotechs Asterias and Capricor have stem cell trials supported by 20+ million in CIRM funding each and have been hitting milestones. These trials are progressing and so far have good safety profiles. Asterias and CIRM have mentioned some possibly encouraging early hints at efficacy as well in its trial, and apparently there are hopeful hints from the Capricor trial too. See …Read More

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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 was based on very limited, unclear data and in large part due to pressure from some patient advocates, serve as a warning to those of us hoping the agency …Read More