September 20, 2020

The Niche

Knoepfler lab stem cell blog

AST-OPC1

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

Australian stem cell biotech Mesoblast announced that it has received regenerative medicine advanced therapy (RMAT) designation from the U.S. FDA. This is very good news for the company and an encouraging development for the field. Interestingly, last month the FDA clarified that there is expanded RMAT designation that can include gene therapies too. At the Meeting on the Mesa in October, an FDA official reportedly presented data indicating that about 1/3 applications for RMATs were granted (see image below from Twitter). This recently announced RMAT …Read More

3 min read

Stem cell good news time…and more will be coming later this week. The stem cell and regenerative medicine world sometimes feels chaotic because so much is going on both on the positive and negative sides of things. There is a whirlwind of activity and developments, and a tendency sometimes within our arena to just avoid bad or complicated news. One mission of this blog is to try to cover the full spectrum of developments including difficult situations, but I also want to focus in …Read More

2 min read

In the whirlwind that is the stem cell and regenerative medicine world, there are many concerning things that need attention, but also good stuff happens too and this post focuses on the positive. The Asterias spinal cord injury clinical trial, a phase 1/2a trial called SCiStar, continues to make encouraging news with a clean safety profile and additional hints at possible positive indicators of efficacy. With the usual, important caveats such as that this is early and it is not an RCT, the SCiStar momentum …Read More