biotechs Archives - The Niche

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

November 8, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

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 […]

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

November 15, 2023 / Professor Paul Knoepfler, Ph.D. / 5 Comments

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

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Weekly reads: Sarepta, epigenomics, FDA on eyedrops

April 16, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

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

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In big shift Dr. Robert Lanza exits Astellas

July 28, 2022 / Professor Paul Knoepfler, Ph.D. / 6 Comments

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

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

July 26, 2022 / Professor Paul Knoepfler, Ph.D. / 2 Comments

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

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

May 6, 2022 / Professor Paul Knoepfler, Ph.D. / 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

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Mayo Clinic charges patients just to talk about clinical trials?

February 15, 2022 / Professor Paul Knoepfler, Ph.D. / 7 Comments

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

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All about Altos Labs and $3B cellular rejuvenation push

January 20, 2022 / Professor Paul Knoepfler, Ph.D. / 2 Comments

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

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

October 19, 2021 / Professor Paul Knoepfler, Ph.D. / 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

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Bluebird bio sickle cell gene therapy trial halted on cancer worry

February 16, 2021 / Professor Paul Knoepfler, Ph.D. / 2 Comments

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

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