The FDA has a unique mechanism called RMAT or regenerative medicine advanced therapy designation. It’s intended to move investigational regenerative therapies forward more effectively. RMAT data I recently updated The Niche RMAT list and it has jumped to 85. The FDA has approved many new RMATs this year. The data on how many applications the FDA […]
The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development. 2 FDA-approved gene therapies for sickle cell Friday’s news follows on recent UK approval for
Let’s start this week’s digest with a discussion of clonal expansion and mutations during cell and gene therapy development. Here’s the article that brought this to mind. Sickle cell gene therapy process may cause cancer-linked mutations in blood stem cells, Fierce Biotech. Clonal expansion This is a story about a non-CRISPR gene therapy using a
Some folks can view data from early, small clinical trials too skeptically or overly enthusiastically, and maybe that’s going on with some preliminary results from Verve Therapeutics. Good news? Bad news? I’m going to start with two articles about the same news that have very different vibes. What do I think? On the whole, I
Weekly reads: Verve Therapeutics, Casgevy UK OK, GDNF, MYC Read More »
Cell therapy companies face many hurdles. I wrote recently about four cell therapy biotechs that have been really struggling. Blogging here at The Niche for more than a dozen years, I’ve seen many companies even go under. That’s bad news for the field, for patients, and for investors. Today I wanted to highlight some cell therapy
3 cell therapy companies doing well: Vertex, Vericel, & Regeneron Read More »
What’s more important than Woolly Mammoth de-extinction research in the stem cell arena? Only maybe a 10,000 other things. Still, the mammoth de-extinction efforts capture people’s attention much more than the average research story. Mammoth De-extinction update Is de-extinction only a pipette dream? This startup has a big, expensive plan to find out, Popular Science.
It feels like we are marching towards a future in which key aspects of human reproduction, including the use of artificial wombs, could be substantially different than for most of history. The FDA is considering allowing a clinical trial for use of artificial wombs in people. Human trials of artificial wombs could start soon. Here’s
Weekly reads: artificial wombs, mice with rat brains, Neuralink Read More »
The New York Times just broke a story on Burst Biologics and Weill Cornell that fits with long-standing troubling questions about regenerative biologics producers. The story also bears on the doctors and hospitals like Weill Cornell that were or are customers of the suppliers. Further, what is the FDA’s role in all of this? The
Burst Biologics unapproved biologic used by Weill Cornell: was there a cover-up? Read More »
Several groups including Viviane Tabar who is a founding scientist at BlueRock Therapeutics are moving forward on potential cell therapies for Parkinson’s Disease. BlueRock just released some new data that’s worth a look. Check out the new BlueRock poster. In today’s post, I briefly discuss the new data. I also go into the larger context
Small positive step for BlueRock Therapeutics Stem Cell Therapy for Parkinson’s Read More »
Many avenues are being pursued to get stem cell therapy to the clinic for eye conditions. These different approaches employ a variety of stem cell types. In some cases, such as with iPS cells, the stem cells themselves are not transplanted. Instead, the iPS cells are used to make differentiated eye cells, such as RPE
Weekly reads: stem cell therapy for eyes research, DMD, cheating death, cancer sensors Read More »