Today the FDA approved 2 gene therapies to treat sickle cell disease, including the first CRISPR-based therapy.

Bluebird Bio, who has the ~$1M more expensive and not-CRISPR therapy, has a single card left to play in this competition: It's been conducting a year-long dress rehearsal for this moment

https://www.statnews.com/2023/12/08/lyfgenia-sickle-cell-gene-therapy-bluebird-vertex-crispr-casgevy/

#health #healthcare #medicine #biotech #pharma #crispr #genetherapy #geneediting #scd #SickleCell #casgevy #lyfgenia #zynteglo

The Food and Drug Administration on Friday approved a powerful 👉treatment for 🔸sickle cell disease🔸, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
The therapy, called "Casgevy", from Vertex Pharmaceuticals and CRISPR Therapeutics, is the ⚠️first medicine to be approved in the United States that uses the gene-editing tool 🔹CRISPR🔹, which won its inventors the Nobel Prize in chemistry in 2020.

The approval marks the first of two potential breakthroughs for the inherited blood disorder.

The FDA on Friday also approved a 👉second treatment for sickle cell disease, called "Lyfgenia", a gene therapy from drugmaker Bluebird Bio.
Both treatments work by genetically modifying a patient’s own stem cells.

Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries the risk of rejection by the immune system, in addition to the difficult process of finding a matching donor.
#casgevy #lyfgenia #crisper #Sicklecelldisease #sicklecell
https://www.nbcnews.com/health/health-news/fda-approves-cure-sickle-cell-disease-first-treatment-use-gene-editing-rcna127979