I’ve been trying to dig out from all the stuff that piled up while I was working toward a big grant deadline last week, catch up on recent news and papers, and during this time I was excited to see the news about an approved gene therapy for a rare form of deafness.
Let’s start with that good news. There’s an interesting twist.
Gene therapy for rare form of deafness
Here’s some coverage: New Gene Therapy Enables Children With a Rare Form of Deafness to Hear, NYT. A key question that teams have to address with approaches to different kinds of deafness is whether the hair cells in the inner ear are intact. If they are, gene therapy becomes relatively less complex but still challenging. The hearing loss in this case is called otoferlin deafness with intact hair cells.
The twist here? The therapy will be free.
“The maker of the therapy, Regeneron, plans to provide it free to any child who needs it. “We wanted to make a statement,” Dr. George Yancopoulos, Regeneron’s chief scientific officer said on Thursday morning.”
It’s important to note that some in the deafness community don’t feel that the condition needs treatment. They view it as a difference more than a health condition.
Cost of deafness treatment: free in this case
There’s been talk over whether some of biotechs are unhappy with the free part of the gene therapy discussed above as it could set a precedent.
The price tags on cell and gene therapies is a hot topic with some being in the millions per dose.
So, looking ahead, envisioning many new gene therapies for hearing loss, what will be the typical price tag for a gene therapy for deafness in 5-10 years? We can be pretty sure it’ll be huge in most cases.
Trials are also testing cell therapies for deafness as well, which is another promising area. Particularly for those cases where the hair cells are lost or damaged.
Longevity, regeneration, grumpy David Sinclair
- OSK-mediated partial reprogramming induces cardiomyocyte dedifferentiation, overcomes cytokinesis barriers, and promotes post-MI endogenous cardiac regeneration, Journal of Molecular and Cellular Cardiology. I think I saw David Sinclair post about this article first. While he seemed excited, I’m less convinced that this is a huge deal, although it is interesting. Note that there was also this important earlier npj regenerative medicine paper by Hina Chaudhry, Cyclin A2 induces cytokinesis in human adult cardiomyocytes and drives reprogramming in mice.
- Longevity Science Is Overhyped. But This Research Really Could Change Humanity, NY Times. This piece got some criticism on Twitter (X) for potentially not being balanced enough. For example, Eric Topol called it over the top. I wonder what Sinclair thought of it. Regardless, the typically very upbeat Sinclair has seemed PO’d the last few times I checked his X feed. This includes posts about media coverage. See an example above. Why tag Elon? He tagged Elon in another recent post as well (see image below). Notably, Sinclair reposted an item that alluded to other scientists being a problem. Isn’t it reasonable to push back on what one sees as longevity hype even if it comes from other scientists? I believe so.
More recommended reads
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- FDA commissioner: ‘Smarter,’ real-time clinical trials could transform drug development, STAT.
- eIF4G2-mediated selective translation of chromatin regulators safeguards adult intestinal stem cell identity and differentiation, Cell Stem Cell.
- Did Kennedy just stack the deck on FDA oversight of peptides? It sure looks like it, STAT. My perspectives on what’s going on inside the FDA on peptides. Perhaps it’s no surprise that in some ways it’s really RFK Jr. in charge of the agency on certain decisions.