December 2, 2020

The Niche

Knoepfler lab stem cell blog

jCyte

3 min read

There is always plenty of complicated, mixed-bag news on the stem cell and regenerative medicine front so it’s great to highlight when there’s some good news out there. Today’s post includes several such upbeat items. At the bottom I’ve included a list of some recent interesting publications as well. Good news on vision front: jCyte Therapy Deal with Santen Pharmaceutical. David Jensen over at California Stem Cell Report reported on this blockbuster deal for a quarter of a billion dollars. Jensen wrote: “The California …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

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

2 min read

The 21st Century Cures Act has some important regenerative medicine language in it. One part of that was attempting to accelerate FDA review of promising investigation regenerative medicine therapies. The mechanism for this was a new designation called Regenerative Advanced Therapy, now renamed Regenerative Medicine Advanced Therapy (RMAT) Designation. I don’t think anyone was sure how this would play out in terms of how quickly the FDA would give RMAT designations and to how many investigational products. Already it’s clear the FDA seems to …Read More