It was exciting when the combined cell and gene therapies Casgevy and Lyfgenia were approved for sickle cell in late 2023.
Still, one of the anticipated challenges was that the therapies could be so expensive that it would limit access. However, since the approval another challenge has emerged for Casgevy. We’ll start there, but first check out the second”Ask a stem cell scientist” video further down in the post from the Canadian student Parmin Sedigh and me. Parmin has made many great videos.
Casgevy challenge
Here’s the news on Casgevy: Vertex’s CRISPR treatment for sickle cell disease hits unexpected roadblock. Vertex’s Casgevy can essentially cure sickle cell patients, but problems collecting stem cells are slowing uptake and threatening its edge, STAT.
From the piece, “The company is also competing with Genetix — formerly known as Bluebird Bio — which had a different gene therapy, Lyfgenia, approved at the same time. Although the pathology of sickle cell disease can make collecting cells from any patient difficult, some doctors say they’ve encountered fewer challenges with Genetix. The company uses a gene-replacement technology that is gentler on cells and thus requires fewer of them.”
More recommended reads
- Cellular survivorship bias as a mechanistic driver of muscle stem cell aging, Science. It’s interesting that NDRG1 (N-myc downregulated gene 1) is involved here. Other work has shown more generally that exercise is the key if you want to fight muscle aging, but people always hope for a pill too.
- Epstein files reveal deeper ties to scientists than previously known, Nature. Stay tuned for more on this. A lot of troubling stuff in the new doc dump.
- Supreme Court Bars Stem Cell Therapy For Autism: Why The Ruling Matters, NDTV. This story is out of India. “The Supreme Court has ruled that stem cell therapy cannot be offered as a treatment for Autism Spectrum Disorder, calling its use outside approved clinical trials unethical and medical malpractice.” Tough words there.
- The role of PubPeer in retractions of highly-cited articles, Dorothy Bishop. This is an interesting analysis that supports the value of PubPeer in the integrity of the scientific literature.