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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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ViaCyte & CRISPR Therapeutics team up to battle diabetes

September 17, 2018 / Professor Paul Knoepfler, Ph.D. / 2 Comments

What happens when one of the most exciting stem cell biotechs ViaCyte teams up with a firm like CRISPR Therapeutics to use a combination of gene editing and cell therapy? Hopefully a synergistic partnership emerges that in this case can develop an effective stem cell-based therapy for diabetes. You can see a press release on

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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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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: FDA OKs 2 gene therapies for sickle cell, new Paolo Macchiarini series, Fujifilm

December 10, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Marie Tornyenu, Casgevy gene therapies for sickle cell

The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development. 2 FDA-approved gene therapies for sickle cell Friday’s news follows on recent UK approval for

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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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Weekly reads: Mammoth De-extinction update, space babies, Alzheimer’s, MSCs don’t help knees

November 5, 2023 / Professor Paul Knoepfler, Ph.D. / 6 Comments

What’s more important than Woolly Mammoth de-extinction research in the stem cell arena? Only maybe a 10,000 other things. Still, the mammoth de-extinction efforts capture people’s attention much more than the average research story. Mammoth De-extinction update Is de-extinction only a pipette dream? This startup has a big, expensive plan to find out, Popular Science.

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Weekly reads: CRISPR sickle cell, Parkinson’s, pig-human chimera concerns

September 10, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

CRISPR gene editing has made rapid progress heading from bench to bedside. Perhaps the fastest has been its progress toward clinical use to combat sickle cell disease. We’ll start with a new paper on one major effort here. CRISPR gene editing. This process often involves cutting DNA, which then can be used as an opening to

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Weekly reads: reprogramming hearing loss, heart disease, eye drops, sickle cell

April 30, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

It’s mostly been a week of good and encouraging news in the regenerative medicine space including with gene therapies maybe with the exception of some eye drops warnings (more below). There’s realistic hope for an approved sickle cell disease soon. I also see some long-term positive news on hearing loss research. Hematopoietic stem cell transplant

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2023 stem cell & regenerative medicine predictions

December 28, 2022 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Toward the end of each year, I make stem cell & regenerative medicine predictions for the coming year. It’s a fun tradition here on The Niche that goes way back. The stem cell field has changed dramatically as reflected in each year’s predictions. Here’s a list of past predictions with my grades of how they

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