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 […]
CRISPR gene editing has made rapid progress heading from bench to bedside. Perhaps the fastest has been its progress toward clinical use to combat sickle cell disease. We’ll start with a new paper on one major effort here. CRISPR gene editing. This process often involves cutting DNA, which then can be used as an opening to
Weekly reads: CRISPR sickle cell, Parkinson’s, pig-human chimera concerns Read More »
It’s mostly been a week of good and encouraging news in the regenerative medicine space including with gene therapies maybe with the exception of some eye drops warnings (more below). There’s realistic hope for an approved sickle cell disease soon. I also see some long-term positive news on hearing loss research. Hematopoietic stem cell transplant
Weekly reads: reprogramming hearing loss, heart disease, eye drops, sickle cell Read More »
Let’s start with some stem cell good news on evolving approaches to treat sickle cell disease involving stem cells. Then on another related front things were more concerning. Stem cell good news on sickle cell disease, a complication Sickle Cell Disease News reports Adding Briquilimab Boosts Success of Sickle Cell Stem Cell Transplant. This is a
Weekly reads: stem cell good news, bad news on sickle cell, MS, IPSCs Read More »
As both a scientist who works on stem cells, cancer, and CRISPR, and a research advocate I’ve been fortunate to meet many patient advocates over the years. Some have been participants in clinical trials themselves. Benefits & risks of clinical trial participation It is very sobering to find out that a clinical trial participant has
Weekly reads: death in CRISPR trial, sickle cell, nose picking & dementia, epilepsy Read More »
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 »
What are the most significant stem cell and regenerative medicine good news stories of 2024? Since it has been an eventful year, there are many possibilities. Please see our poll at the bottom of this post and weigh in on what you think is the most important encouraging news. If you don’t see your choice
Poll on stem cell & regenerative medicine good news of 2024 Read More »
It’s always fun to see startups with cool ideas in the regenerative medicine space and a new one called GC Therapeutics got some nice coverage this week. What do you think the name of this firm stands for? I have one main idea I mention below. Before we jump into it, I want to introduce