The biotech Sarepta has had a complicated go of it with the FDA sometimes related to their Duchenne muscular dystrophy (DMD) gene therapy efforts. In late 2016 I wrote about how there was some controversy as the FDA approved the Sarepta drug eteplirsen (Exondys 51) also for DMD, going against an advisory panel that had …
Weekly reads: Sarepta, epigenomics, FDA on eyedrops Read More »
The recent news of Vertex Pharma buying small stem cell biotech ViaCyte and Doug Melton moving to Vertex brought to mind an old stem cell biotech called Advanced Cell Technology (ACT) and its leader, stem cell biologist Dr. Robert Lanza. Years ago, big pharma firm Astellas bought ACT and Lanza moved to be a leader of regenerative …
When I’m asked about the most promising areas of stem cell clinical research, cell therapy for type I diabetes is near the top including work by a biotech called ViaCyte. Other firms are in the mix too, boosting the overall odds of success in the coming decade. It’s really that promising even though there are …
ViaCyte purchase puts Vertex in driver’s seat on stem cells for diabetes Read More »
If there’s one area of biologics where the FDA has been consistently busy lately it’s the perinatal cell therapy space. The agency has made it clear in the last year that allogeneic birth-related cell products are often drugs requiring premarket approval. This has mainly manifested at a practical enforcement level. We’ve seen actions taken in …
End of FDA “grace period” impacted perinatal cell therapy biotechs Read More »
What if some legit place like The Mayo Clinic charged people just for the opportunity to talk to a doctor about possibly enrolling in a clinical trial? Imagine this kind of conversation. “Hey, Doc, I have this medical problem and heard you have a relevant clinical trial. Tell me about it.” Then the doctor fills …
Mayo Clinic charges patients just to talk about clinical trials? Read More »
I wrote briefly a few months ago about Altos Labs. It’s a new kind of biotech institute that is focused on cellular reprogramming. Now we know much more and it’s clear this is a huge deal. What’s in this article What is Altos Labs? | Cellular rejuvenation | Reprogramming oldness | Challenges | Altos Strategies | Analysis of …
All about Altos Labs & its $3B cellular rejuvenation push Read More »
It’s hard in the cell therapy field not to get excited even with bits of seemingly encouraging news but with N=1 reports we have to be very cautious and that’s the case with news from Vertex on their 1st stem cell therapy for diabetes trial participant. Vertex stem cell therapy for diabetes VX-880: N=1 This …
Vertex upbeat on 1st stem cell therapy for diabetes trial participant Read More »
Recently I was going through a list I’m updating of regenerative medicine biotech companies including Bluebird bio. Now just a few days later the world seems turned upside down for the firm and maybe to some degree for other companies doing related work. Bluebird bio blues: 2 trial participants get cancer News broke today that …
Bluebird bio sickle cell gene therapy trial halted on cancer worry Read More »
Graft-versus-host disease (GVHD) is counterintuitive In the weeks and months following a transplant, a major concern is the recipient’s immune system rejecting the “foreign” biological material. But in GVHD, the opposite happens: transplanted tissue unleashes a horde of T cells that spark a cascade of inflammation, within 100 days. Typically, GVHD follows a bone marrow …
Mesoblast MSCs Quell Peds GvHD; On Road to FDA Approval? Read More »
Today’s post is the latest edition of my weekly recommended reads, which this week include Fate Therapeutics, COVID-19 updates, AAMC, and more including some great pubs such as one on 2 modes of PRC2 function. Also check out this handy resource: Helpful 2020 List of Stem Cell Journals and this blast from the past post of 10 …
Recommended reads: COVID-19 updates, FDA stem cell nod, PRC2, more Read More »