<
https://web.archive.org/web/20231108195733/https://www.nytimes.com/2023/10/31/health/sickle-cell-fda-cure-crispr.html>
"A panel of experts said on Tuesday that a groundbreaking treatment for sickle
cell disease was safe enough for clinical use, setting the stage for likely
federal approval by Dec. 8 of a powerful potential cure for an illness that
afflicts more than 100,000 Americans.
The Food and Drug Administration had previously found that the treatment, known
as exa-cel and jointly developed by Vertex Pharmaceuticals of Boston and CRISPR
Therapeutics of Switzerland, was effective. The panel’s conclusion on Tuesday
about exa-cel’s safety sends it to the F.D.A. for a decision on greenlighting
it for broad patient use.
Exa-cel frees patients from the debilitating and painful effects of this
chronic, deadly disease. If approved, the Vertex product would be the first
medicine to treat a genetic disease with the CRISPR gene-editing technique.
It could also be the first of a series of new options to cure the excruciating
illness. By Dec. 20, the F.D.A. will decide on a second potential cure for
sickle cell, a gene therapy devised by the company Bluebird Bio of Somerville,
Mass."
Via
Future Crunch:
<
https://futurecrunch.com/good-news-rail-education-ethiopia-conservation-congo/>
Cheers,
*** Xanni ***
--
mailto:xanni@xanadu.net Andrew Pam
http://xanadu.com.au/ Chief Scientist, Xanadu
https://glasswings.com.au/ Partner, Glass Wings
https://sericyb.com.au/ Manager, Serious Cybernetics
