Today’s post includes a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT. Sometimes people call these “r mat” with a space. I also include FDA data on RMAT submissions via an FDA RMAT resource page. At this point as of May 2025, I have found […]
Updated FDA granted RMAT List (130) Read More »
What is the future of Genome Medicine? The meeting by that same name that I’m at down here in La Jolla is all about tackling this question and the line up of speakers today on the first day is amazing. I’m speaking about IPS cells as a basis for personalized medicine tomorrow morning so that’s exciting.
Live blogging Future of Genome Medicine: great talks by Feng Zhang & others Read More »
Editor’s note. Caroline Simons attended both the April 28th (see her report on that here) and 29th Paris meetings on human gene editing/genetic modification. Today, we have her in depth report on the April 29th meeting. I have posted her piece in full with only minor edits. If you are in a rush you can
In Depth Report from April 29th Paris Human Gene Editing Meeting Read More »
Change is in the air for the California stem cell agency, CIRM. It has a new President, Randy Mills, and CIRM is soon going to be like a new agency too. I recently did an interview with Mills that illuminates some of the big changes that are imminent. I also asked him what his favorite
Radical Vision for CIRM Future: Interview with New Prez Mills Read More »
The FDA alerted the public about issues related to amniotic eye drops last year and now the agency just warned the manufacturer of one such product called Regener-Eyes. The manufacturer is Regenerative Processing Plant, LLC. The warning letter was addressed to its owner, Carl R. Harrell, MD. What’s the deal with this warning? Let’s start
Regener-Eyes maker gets FDA warning on amniotic eye drops Read More »
Earlier this year Chinese researcher He Jiankui finished his prison sentence. When I saw that news I wondered about the next chapter for him. It seems he’s already back in the lab doing genetics research. At least that’s according to a blog by He Jiankui himself. Can we trust that what he has written on his website is
He Jiankui says he’s back in the lab after prison for CRISPR babies Read More »
I get a lot of questions about stem cells for ALS and more recently people have been specifically interested in NurOwn from BrainStorm Cell Therapeutics. Recently, the FDA denied BrainStorm’s request for a BLA for NurOwn. The company felt its Phase III trial was encouraging. It sounds like they will move forward with a follow-up
Weekly reads: NurOwn, key gene screen, MS, Croce mess Read More »
Magenta Therapeutics is a biotech company with a portfolio including stem cell products. Their most well known investigational drug product is an expanded umbilical cord stem cell product called MGTA-456, an “ex-Novartis” product. Magenta just released new trial data on MGTA-456, which I see as a case of good news-bad news, and investors seem to
Magenta Therapeutics Good News-Bad News Trial Data Read More »
It’s germline, heritable human CRISPR time, right? Wrong. But the particularly enthusiastic supporters of heritable human CRISPR often cite hypothetical benefits in glowing terms, but either don’t mention risks or strongly downplay them. These fans also tend to leave alternative, proven and safe technologies such as preimplantation genetic diagnosis (PGD) out of the discussion or
Countering that Pro-Heritable Human CRISPR WSJ Piece Read More »