September 18, 2020

The Niche

Knoepfler lab stem cell blog

regenerative medicine advanced therapy (RMAT)

3 min read

It’s notable how the FDA now considers gene-editing a kind of regenerative medicine. This means that various gene therapy products in development technically qualify as regenerative therapies. Ever since the agency began its rapidly growing regenerative medicine advanced therapy (RMAT) designation program, we’ve seen an increasing number of gene therapy biotechs and products qualify as RMATs. As a result, the RMAT-qualified gene therapies get to go into a speedier lane of FDA oversight. Today’s post is a mix of recommended reads in the regenerative …Read More

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

3 min read

The year of 2018 has already been a wild one for stem cell news. There are many developments on a variety of fronts. Here are some of the most notable news bites of the last month or so. RMAT Wave. The FDA continues its warp-speed issuance of Regenerative Medicine Advanced Therapy (RMAT) designations with 20 such designations now in the public domain with Cellerant Therapeutics’ announcement and possibly a few more not yet out there. I wrote about the “carrot” of RMATs as relative …Read More

3 min read

The Japan stem cell and regenerative medicine oversight system has been designed to move investigational therapies forward fast, much faster than the systems in other countries such as the U.S. But how well is it working so far? It’s an important question both for that country and more globally as the Japanese system was touted by some as a model and a reason why the U.S., to remain more competitive in this industry. Some of the same folks felt that the U.S. should also …Read More