Are you a risk-taker or do you do everything possible to avoid risks? Taking risks in science is necessary, but is there a wise way to take the risks you do? Science and medicine need a certain amount of risk and risk-takers to make transformative leaps forward. The risks in biomedical science can be conceptual […]
Some thoughts on taking risks in science & medicine Read More »
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 »
The news that CRISPR-Cas9 gene editing in its current form may not work in a substantial fraction of people due to many of us having immunity to Cas9 came as a shock to many, but if you think about it, maybe it’s not so surprising. I don’t see it as the end of the world. A (preprint) from
Keep calm & CRISPR on: perspectives on report of human Cas9 immunity Read More »
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
Mesoblast gets FDA RMAT; List of 10 total designations so far Read More »
The California Institute for Regenerative Medicine, more widely known as CIRM, has accomplished big things over the course of its history of about a decade and sparked a great deal of innovation, but what does the future hold for our stem cell agency? CIRM has a new, respected President and CEO Maria Millan solving one
Update on CIRM future: will it include 2020 measure? Read More »
By Samantha Yammine When our TV stops working, it’s usually because one of the wires has come unplugged. We begrudgingly huff and puff over to the back of the TV, track down the loose culprit, plug it back in, and boom: back to Netflix. If you multiply all those wires in the back of your
I started The Niche blog in largely its current form early in 2010, and now 7+ years later we’ve hit two big milestones recently. First, about a month ago, we reached 10,000 comments made on the blog. Thanks for being involved in the discussion on the Niche blog! Second, we reached 7,000,000 total page views
Two more big milestones for The Niche blog Read More »
Grad school…would you do it all over again if you could jump in a time machine? It’s hard to imagine, but it was 24 years ago that I started at UCSD as a graduate student. Think how different the world was in 1993. Now almost a quarter century later, the world of science is very
Twenty years later: would this dataholic still go to grad school? Read More »
Human germline CRISPR raises major bioethical considerations, but what about technical issues? Setting aside the many ethical issue about the general idea of human modification itself, could this really work? Yes in theory it could, but there are some very tough technological challenges that could and likely would cause failures or unacceptable outcomes at many steps
7 tech hurdles to human germline CRISPR Read More »
Some notable newly published Phase II data from the Duke cord blood for cerebral palsy trial unfortunately doesn’t give much reason for optimism that this approach is going to have a substantial positive effect for these kids. The double-blinded, placebo-controlled study did not find a meaningful benefit overall from infusions of autologous cord blood (ACB) for
Cord blood for cerebral palsy: mostly discouraging new trial data Read More »