The FDA has been busy with the COVID pandemic but it has many other things on its plate as well including cell therapies. Today I’m sharing some recent agency news and brief perspectives on it. FDA focus on clinic-related umbilical cord cell drug makers The agency warned an Idaho company that produces and sells umbilical …
FDA news: warning letter pattern, cancer moonshot, CAR-T & gene-editing guidance Read More »
The paper of the week reports using base-editing, a kind of gene-editing, to reverse mutations associated with rapid aging syndromes, generally called progeria, but there are a lot of other interesting pubs to recommend for reading this week. I go over it all in this post. Gene-editing to fight premature aging syndromes In vivo base …
Weekly reads & paper of the week: gene-editing vs. aging Read More »
As a pivotal advance in the gene-editing field and timeline, CRISPR continues to be utilized for research on stem cells and human diseases. Today, I will explain some of the most notable recent findings in the stem cell-CRISPR field. To start things off, I have also created an infographic that briefly explains what CRISPR-Cas9 is, …
New research on CRISPR gene-editing in stem cells, infographic Read More »
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 …
Weekly reads: Vertex & CRISPR Therapeutics, Arnold Caplan death, MS genetics Read More »
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 …
20 stem cell & regenerative medicine predictions for 2024 Read More »
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 …
2023 stem cell predictions grades reflect wild year for regenerative medicine Read More »
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 …
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 …
It wasn’t that long ago that Chinese scientist He Jiankui got out of jail. Why was he in there? For doing unauthorized human CRISPR to make heritably gene-edited humans. He botched that and apparently forged some approvals to make it happen. The three children involved may have life-long health problems as a result. I wrote …
Totipotency literally means all powerful, but it refers in biology to specific cells. These cells can make every type of cell in the body of an organism plus the extraembryonic tissues needed for development. This includes humans. So if you could reprogram human cells like blood or skin cells into totipotent stem cells, you might …
Weekly reads: gene therapy nod, Nature pub ethics, CRISPR LDL Read More »