June 3, 2020

The Niche

Knoepfler lab stem cell blog

FDA 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

4 min read

Every year I look into my stem cell crystal ball and make predictions for our field including regenerative medicine. Well, I don’t really look into a crystal ball, but I try to use a combination of what I’m hearing through the grapevine, stuff I’m reading, some info that is not yet in the public domain, and common sense to make predictions. Today’s post has my 20 predictions for next year, 2018. You can see my 2017 predictions and my grading of how I did here. …Read More

3 min read

It’s a difficult, but very important road to conclusively prove that an investigational stem cell therapy is both safe and effective in order to get final approval FDA or equivalents in other countries. For this reason, it is beneficial to highlight even small, but positive steps forward including encouraging pre-clinical data. Today I’m doing a review of stem cell therapy good news in the clinical path with a focus on biotechs in particular (in alphabetical order), but starting first with news from here at …Read More

3 min read

Can a stem cell clinic business get FDA RMAT designation? At least one announced it was trying, but now seems to have given up. Stem cell clinic business US Stem Cell, Inc. has reportedly announced that it is at least temporarily abandoning its efforts at getting Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The company’s penny stock $USRM has been gyrating for months and I had earlier wondered if could be some fake news about it. The USRM news now seems real and not …Read More