I have been reading up on something called the Los Angeles Project that involves gene editing embryos to try to make new animals. Biohacker Josie Zayner and Cathy Tie are the founders. They even have aspirations to make dragons and unicorns with CRISPR. That resonated with me for reasons I’ll talk about in a minute. What’s […]
The cell therapy biotech Capricor recently released new data from their Duchenne Muscular Dystrophy (DMD) trial work. The data have generated excitement in the patient advocacy community. The stock has skyrocketed. How exciting are these new data? CEO Linda Marbán is quite upbeat and I think it’s justified. The firm is now seeking FDA approval
Capricor seeks FDA approval of deramiocel for Duchenne based on encouraging data 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 »
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
In big shift Dr. Robert Lanza exits Astellas Read More »
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 and $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 »