I’ve been following the Huntington’s disease space carefully for about 15 years. I always hoped that some kind of promising gene therapy might emerge. Stem cell therapies or combination cell-gene therapies could have promise for Huntington’s too. Over the years, I got to know Judy Roberson, a wonderful HD patient advocate.
I’m going to start this week’s wrap of regenerative medicine research with an FDA decision on HD. Patient advocates view this with hope.
FDA clears uniQure to submit Huntington’s treatment
Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval, STAT. This is another big FDA reversal. It wasn’t so long ago that the agency didn’t seem so upbeat on UniQure’s data or approach…or something. Of course, that was back when Prasad and Makary were the agency leaders.
The gene therapy, AMT-130, is a one-time AAV5 product that encodes a designer microRNA that targets HTT mRNA. The FDA now says phase 1/2 data on AMT-130 can be used for the application. I wouldn’t be surprised if the agency gave an accelerated approval for AMT-130 even without phase 3 data given the pressing need for new HD approaches.
Then, uniQure would do a confirmatory trial.
Here’s more HD news, but on the investigational cell therapy front: First participant dosed in pioneering neural stem cell trial for Huntington’s disease, HD Buzz.
More recommended reads
- Marjorie Taylor Greene i’m trying to filibuster aging with stem cells!!! TMZ. Of course, she did.
- Scientists see promise in NIH proposal to cap number of grants they receive, STAT. I support the idea of a cap here. It’s counterproductive that some PIs have 6 or 7 R01s as it’s been shown that productivity per unit of funding decreases substantially when so much funding is concentrated in one lab.
- US public support for human ES cell research continues strong at 63%. I happened to look up this polling recently.
- Human Cell Atlas leader’s tie to 10x Genomics raises conflict-of-interest questions, STAT.
- The Rise of Eyes Began With Just One, NYT. Did vertebrates start as cyclopses?