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Weekly reads: Vertex, stem cells for MS, Athersys, 900-day paper review at Nature

March 3, 2024 / Professor Paul Knoepfler, Ph.D. / 1 Comment

“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.

Vertex Pharmaceuticals has a lot going for it right now on the cell therapy front. For instance, they have the strongest type 1 diabetes cell therapy pipeline after some recent acquisitions. Still it’s not a simple matter to succeed in the cell therapy space even with one therapy for one targeted disease. Trials are tough […]

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Something Elon Musk & I agree on: pushing back on anti-aging bluster

February 28, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Would you guess that Elon Musk might be into using biotechnology in a high-profile way to try to live far longer than usual, say to age 150? That would have been my assumption. There are countless things about Elon Musk that I really don’t like. I also don’t see many issues on which we are

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Weekly reads: Xist & autoimmune disease in women, Crohn’s disease, dumb headline of the week

February 4, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Readers of The Niche have asked me many questions about stem cells for autoimmune disease but the puzzle of why women get these conditions more often than men hasn’t come up before here. For instance, why is MS so much more common in women than men? It’s remained somewhat of a mystery over the years.

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Long-term hope on stem cells for deafness

February 1, 2024 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

A reader of The Niche recently asked me, “What about stem cells for deafness?” It’s a great question and, as they pointed out, stem cells for vision loss gets much more attention. I’ve covered the translational progress of stem cells to treat many diseases but there has been less progress for deafness in the regenerative

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Weekly reads: Vertex & CRISPR Therapeutics, Arnold Caplan death, MS genetics

January 14, 2024 / Professor Paul Knoepfler, Ph.D. / 5 Comments

The biotechs Vertex and CRISPR Therapeutics have an interesting relationship as biotechs. They are partners are multiple levels but also are very different as companies including in size. There’s been a key development in one of their partnerships. Before we jump into that, please check out the video version of my 20 stem cell and

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20 stem cell & regenerative medicine predictions for 2024

January 3, 2024 / Professor Paul Knoepfler, Ph.D. / 4 Comments

Each year I make predictions for the stem cell and regenerative medicine field for the following year. Today’s prediction post is focused on what to expect in 2024. You can also see my stem cell predictions for 2023 and my grades on how I did. I also have made a video version of these predictions so please

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2023 stem cell predictions grades reflect wild year for regenerative medicine

December 27, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Every year I make stem cell and regenerative medicine predictions. Looking at my predictions for 2023, they reflect a wild year but in many ways a good one overall. Below I have graded my 2023 predictions. Overall, my crystal ball gave solid results. Some of the predictions have been condensed to keep things concise, but you

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Holiday reads: woolly dog vs. mammoth, man eggs, designer B-cells, bluebird bio blues

December 24, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

What is a woolly dog? Is it somehow related to the Woolly Mammoth? Before we get into that and the rest of my recommended reads, note that I’m finalizing my list of contenders for this year’s The Screamers Science Hype Awards. Last year The BBC won The Screamers for an article “Baby’s life ‘probably saved’

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Weekly reads: clonal expansion, Golden Retriever cancer, FTC hits genetics firm on testi-phony-als

November 26, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Let’s start this week’s digest with a discussion of clonal expansion and mutations during cell and gene therapy development. Here’s the article that brought this to mind. Sickle cell gene therapy process may cause cancer-linked mutations in blood stem cells, Fierce Biotech. Clonal expansion This is a story about a non-CRISPR gene therapy using a

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Weekly reads: Verve Therapeutics, Casgevy UK OK, GDNF, MYC

November 19, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Some folks can view data from early, small clinical trials too skeptically or overly enthusiastically, and maybe that’s going on with some preliminary results from Verve Therapeutics. Good news? Bad news? I’m going to start with two articles about the same news that have very different vibes. What do I think? On the whole, I

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