biotechs

Capricor seeks FDA approval of deramiocel for Duchenne based on encouraging data

/ / 1 Comment
Linda Marbán, Capricor

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 »

3 cell therapy companies doing well: Vertex, Vericel, & Regeneron

/ / 5 Comments
Vericel, Cell Therapy Companies

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 »

Weekly reads: Sarepta, epigenomics, FDA on eyedrops

/ / Leave a Comment
Sarepa CEO interview.

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 »

ViaCyte purchase puts Vertex in driver’s seat on stem cells for diabetes

/ / 2 Comments
Doug Melton, Vertex, ViaCyte

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 »

End of FDA “grace period” impacted perinatal cell therapy biotechs

/ / 1 Comment
knot in umbilical cord, perinatal cell therapy

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 »

Mayo Clinic charges patients just to talk about clinical trials?

/ / 7 Comments
The Mayo Clinic.

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 »

All about Altos Labs and $3B cellular rejuvenation push

/ / 2 Comments
Altos Labs PIs

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 »

Vertex upbeat on 1st stem cell therapy for diabetes trial participant

/ / 14 Comments
“While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression,” said Bastiano Sanna, Ph.D., executive vice president and chief of cell and genetic therapies at Vertex.

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 »

Bluebird bio sickle cell gene therapy trial halted on cancer worry

/ / 2 Comments
bluebird bio, sickle cell gene therapy

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 »

Subscribe to Our Newsletter

Be the first to know about the latest developments in stem cell and regenerative medicine research.