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 »
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.
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. As of December 12, 2023 there are 85 RMATs in the public domain. I also include FDA data on RMAT submissions via an FDA RMAT resource
Updated FDA granted RMAT List (85) 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 »