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 …
Is 2022 going to be any better than 2020 or 2021 when it comes to things in general or for biomedical science? Related to the pandemic? It’s too soon to say but if I had to guess right now I’d venture an optimistic view that overall it’s going to be somewhat better by the end …
Weekly science reads: senescence, alt CRISPR, sickle cell update Read More »
Recently I was going through a list I’m updating of regenerative medicine biotech companies including Bluebird bio. Now just a few days later the world seems turned upside down for the firm and maybe to some degree for other companies doing related work. Bluebird bio blues: 2 trial participants get cancer News broke today that …
Bluebird bio sickle cell gene therapy trial halted on cancer worry Read More »
I’m playing catch-up on some reading given how busy I’ve been and this includes a groundbreaking NEJM pub on CRISPR for Sickle Cell and Thalassemia. CRISPR for Sickle Cell From December, here’s the key paper in the NEJM: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. There’s a lot to like about this clinical …
Recommended reads: CRISPR for Sickle Cell, Parkinson’s, & more Read More »