Where do things stand with potential applications of CRISPR and other gene editing technologies in patients? Overall, things are looking very positive.
CRISPR human trials
This week the third big international human genome editing meeting took place in London. The summit addressed numerous potential clinical applications. It was good to see the agenda included quite a few new speakers.
What did the organizers have to say after the meeting concluded?
Here is the official word: Statement from the Organising Committee of the Third International Summit on Human Genome Editing. The statement here generally makes very good sense on the key points. Notably, the more specific statement on germline editing is logical but could have been even stronger. What about He Jiankui? He seems hoping for some comeback and, of course, his actions were reportedly discussed at the meeting. That makes sense. I see him more as part of the past, the history here.
The biggest news here is how promising somatic editing is looking on multiple fronts. CRISPR sickle cell work in particular is very encouraging.
Victoria Gray, who received an investigational gene-editing therapy for sickle cell and is feeling dramatically better since, was one of the speakers at the London meeting. Being a clinical trial participant takes courage. I really admire her.
More of the meeting coverage and related material:
- Why genetic engineering experts are putting a spotlight on Victoria Gray’s case, NPR.
- In wake of gene-edited baby scandal, China sets new ethics rules for human studies, Science.
- Then we have China’s new human gene-editing rules worry experts, BBC. It’s interesting how different headlines can be on the same topic, right?
- Forget designer babies. Here’s how CRISPR is really changing lives, MIT Tech Review. Antonio has a point, except that we can’t forget He Jiankui or risks of other possible heritable stabs at editing. I think a key point is the somatic work deserves more focus.
- More than 200 people have been treated with experimental CRISPR therapies, MIT Tech Review. It’s helpful to see the scope of clinical trials here. However, the use of the word “treated” in the title is not great. The word implies known benefit. Instead of “treated with” I would have said “given”. What do you think? Too nick picky?
- Does Gene Editing Have a Future in Reproductive Medicine? NYT. This guest piece by anthropologist Eben Kirksey is kind of an enigma to me. What’s his point? Are you just making the point that things are very complicated on the heritable front? I asked him on Twitter if he could clarify his main points but didn’t get a reply.
Good news as FDA to review Mesoblast BLA; agency spikes Neuralink
It was surprising good news to see that the FDA has said it will review Mesoblast’s BLA for GvHD. Earlier the agency had rejected it. I’m not entirely clear on what changed. Perhaps there were some key newer data. New data had been mentioned over the past months but it was a little unclear just how new it was.
It’s still early in the year, but I could end up wrong in part of my prediction for 2023 about stem cell biotechs, at least as relates to Mesoblast. There is real hope now that they may ultimately get approval. I’m glad the firm got this upbeat news.
Things are not looking so good for Neuralink. U.S. regulators rejected Elon Musk’s bid to test brain chips in humans, citing safety risks, Reuters. This bad news for Neuralink was one of my stem cell and regenerative medicine predictions for 2023. At least I’ll probably get one right.