June 5, 2020

The Niche

Knoepfler lab stem cell blog

retinitis pigmentosa

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

1 min read

CIRM has reinvented itself in a new form, CIRM 2.0, under the leadership of its new President Randy Mills. CIRM is almost like a stem cell that has changed into another new kind of stem cell. Us scientists could see the change coming in the “programming” at CIRM in terms of how it was operating as the 2.0 version, but what would it bring in practical terms? We are starting to see the new CIRM 2.0 in action and by all signs it looks pretty …Read More