FDA approves first gene therapy for treatment of genetic hearing loss
fda.gov244 points by JeanKage a day ago
244 points by JeanKage a day ago
I'm partially deaf and I've worn hearing aids since around five years old. I used to think it didn't affect me but as I get older I increasingly suspect it's negatively impacted my life more than I thought. Just the additional difficulty understanding in noisy environments affected my social life and development, and who knows what kind of knock-on effect that's had.
Anyway, any progress in treating similar conditions is great news.
They do say that hearing loss in old age can speed degradation, or maybe it is just correlated.
It definitely speeds the effects of dementia and similar because your brain insists on filling in what you didn’t hear and it tends to be wildly negative, at least in my two experiences of having gone through it.
Although this affects a small number of people / kids per year - this is a great development. There's a related article^ which had a very moving account from a mom whose son has received this treatment.
^: https://www.npr.org/2026/04/23/nx-s1-5795526/deafness-gene-t...
I’ve been following this for a personal interest. Decibel Therapeutics was working on this for quite a while with a lot of success before they were acquired by Regeneron and it was very promising but the next ones in the pipeline were personally relevant. They’re a fix for the GJB2 gene mutation that causes hearing loss. Unlike the OTOF error, this one is progressive so you have to get to it fast.
My wife and I carry mutations to the gene so we’ve done preimplantation genetic testing to select the embryos that haven’t been affected and our daughter can hear just fine! We have enough unaffected embryos that we can have another child but if we can have a third we’d probably want a boy[0] and both of our male embryos are coincidentally affected. If somehow we’ve managed to delay long enough for the corresponding Decibel TX AAV.103 gene therapy to come to market, then this will be an incredible triumph of modern science and technology over nature.
Here’s hoping!
If you’re curious about this process, I’ve written about it here: https://wiki.roshangeorge.dev/w/IVF
And here’s the treatment pipeline image I nicked off decibel TX’s website before they were acquired a year and a half ago https://wiki.roshangeorge.dev/w/File:Screenshot_Decibel_Tx_P...
I’m super thrilled everything has gone through so fast.
0: it would just be nice to have children of both genders; a weak preference - if I have 3 daughters I would be thrilled anyway
you can detect sex and defects in embryos now and select for them? Is this expensive?
I thought public policy was avoiding this specific thing for this specific reason, but I’ve been admittedly out of the loop
Yes, and they can do it at six weeks, too. Downs syndrome is the common thing to be avoided that they're testing for.
Sounds like advertisement to me.
But, ignoring this, you wrote:
> If somehow we’ve managed to delay long enough for the corresponding Regeneron AAV.103 gene therapy to come to market, then this will be an incredible triumph of modern science and technology over nature.
How does this relate to offspring exactly? The "delay long enough" part makes no real sense to me. Also, no treatment is usually always 100% effective, so I don't understand the "delay long enough" part either. Plus, there is no "nature" anymore than there is a divine being. What this here is simply the difference between having technology; and not having it. "Nature" is not part of any equation here other than regular genetic information and how it is changed, "naturally".
Haha welp. Just a fan. But if it means anything, I’ve been at it for quite a while. Here’s another comment from years ago https://news.ycombinator.com/item?id=42149228
That’s about when I found out about the Decibel Tx therapy ongoing. If I have any underlying motivation it’s that maybe we could get earlier access to the therapy. Sadly, scientific studies don’t admit fan clubs preferentially.
The delay long enough is that we have all of our embryos in cold storage and we’ve been able to select the ones that are unaffected. My daughter is a bit over a year old and we’re planning a second implantation in the next few months where we will obviously select an unaffected embryo. But a third child, if my wife wishes, will be another 3 years from now. If PGT allowed us to go from a 2023 plan to have kids to a 2029 son born from a male embryo we were able to keep in cold storage till a therapy was available to give him hearing at birth, I think that qualifies as being able to delay long enough.
And about the Nature comment, imagine it a form of metonymy. I’m just referring to random chance and genetic mutation and so on. I’d call my glasses a triumph of man over nature too :)
>Plus, there is no "nature" anymore than there is a divine being. What this here is simply the difference between having technology; and not having it. "Nature" is not part of any equation here other than regular genetic information and how it is changed, "naturally".
the pedantry of some HNers never ceases to amaze me. all of these words to not actually say anything.
This is really cool and awesome for people with this condition!
Unfortunately, I have seen treatments arrive for almost every type of deafness except the one affecting me. I contracted the mumps at a very young age, approximately 5 months old, and I now have nerve deafness in one ear making me completely deaf in that ear. The ear drum still operates, as I feel pain in that ear when around extremely loud noises. I absolutely do not want a cochlear implant, so it seems I may be stuck like this for the rest of my life. :-(
Same situation here. I haven't looked at the state of the art in about 2 years, though. I was told that I was not a candidate for CI since the auditory nerve is damaged. What I found back them was some early research into cell transplantation as neural regenerative therapy, but there wasn't much activity that I could find.
Do you worry about having an increased risk of Alzheimers/Dementia?
No, not really. Why?
There are reports that hearing loss correlates with dementia. I can't evaluate the reports but I'm aware of them.
This is a very cool example of a targeted gene therapy for a very specific type of hearing loss. As the article says it only applies to a subtype of genetic hearing loss which makes up 2-8% of genetic cases, but it’s nice to see such niche therapies being developed and approved despite the smaller number of people who could benefit. It underwent an accelerated review through a new program met to fast track treatments for rare conditions like this which would normally be difficult to get approved due to the small scale.
Coincidentally, I'm reading this at the doctor's office reception, waiting to check my (potentially genetic) hearing loss. Hope it's something simpler, but good to know there are advances in this area.
Very positive news. I have something similar but in a much more difficult gene with different manifestation, but at least this gives me hope that something might come up in 1-2 decades.
I would think the time frame is not nearly as long as that… hopefully
Fingers crossed. I thought the same when I had heard Stefan Heller at Stanford give a talk 15+ years back. I am sure progress is happening but Biology is hard and the hope is AI and other developments are giving it a push
"These kind of genetic therapies seem to reinforce this idea of deafness being a problem in need of eradication, and that the only solution for disabled people to fully assimilate into society is through a medical intervention," says Jaipreet Virdi
This just seems so incredibly stupid to me.There is no winning with some people. If there was no cure, I am going to bet that these same people will be complaining that the pharma industry doesn't value them enough.
Likely because you have no experience with needing accessibility nor the disabled community? There are two approaches, the first usually being followed by non-disabled people:
1. You can only be whole ifwe heal you 2. I am fine as long as the world doesn't make me suffer
I can see both sides, but I also see that 1 is incredibly condescending.
(blind user)
Yes let’s go make deafness optional for adults and eradicate childhood deafness. Zero moral ambiguity.
Consider this from the perspective of a deaf person. While it seems silly from the perspective of a hearing person, a lot of people in the deaf community are concerned that they are viewed as having a problem needing to be fixed, rather than competent, highly functioning people.
Coming from somebody in the half-deaf community, please, medical science, find a cure for the problem that I have that needs to be fixed.
Thank you for saying this so energetically and straightforwardly. To the greatest extent possible, we should endeavour to call things what they are.
At the risk of turning this positive thread negative, there exist deaf people who would deny their children the opportunity to hear, and very little makes me more furious than society's willingness to tolerate this form of child abuse.
Then they can just not get the treatment...?
Part of the concern is that if a cure for deafness becomes standard, then resources for the deaf community (e.g. sign language interpreters) may no longer be available for people who either cannot or choose not to get the treatment.
There's also an issue that, assuming they work similar in this regard to cochlear implants, the treatment has to be performed at a very young age before its possible for someone to consent or choose whether they want to be part of the deaf community.
I understand these arguments but don't find either of them compelling whatsoever.
Here's my test: If my child was deaf and asked me when they were old enough to know that I declined to have them treated based on these arguments, I cannot even imagine them being okay with that.
I understand their perspective but it’s still silly.
I’m a competent, highly functional person. I also have idiopathic hypersomnia and IBS-D. I’d love a fix for either; I want to live the best life possible.
The whole deaf community opposition to treatment reads as just a defensive mechanism. Being deaf means that one of your limited amount of senses doesn’t work. By definition, they’re disabled. That’d be like people whole are really near or farsighted not using glasses because they’ve decided not being able to see is their culture or personality. It’s ridiculous, and that viewpoint should be more than ridiculed when deaf parents don’t pursue treatment for their children.
The difference between deafness and other disabilities is that deafness forces you to communicate differently. That communication difference creates a separate language community, which develops its own culture, just as every other language community does. When people belong to a certain culture, that belonging often forms a part of their sense of self.
When it comes to children, then, the question is not just "do I want my child to hear better than I can", but also "do I want my child to speak the same language and belong to the same culture that I do" - something most parents want very much.
> When it comes to children, then, the question is not just "do I want my child to hear better than I can", but also "do I want my child to speak the same language and belong to the same culture that I do" - something most parents want very much.
That's simply: 'what is best for my child' vs 'what is best for my relationship with my child'. Only one of those actually has the best interests of the child at heart. Only one of those opinions is respectable. Growing up with the latter leads to resentment towards the parent generally.
It’s like raising your kid in a remote cabin without access to services and schools.
Its more like making that choice: and it having it be permanent. The child can never visit the city. You can always make someone deaf (unethical), but you can't always reverse hearing loss at an advanced age.
I think your argument cuts the opposite way that you intended. IIUC, the majority of deaf children are born to non-Deaf parents.
Ok but maybe first we could start ridiculing people who skip infant and child vaccinations, or teach their kids religion.
I wonder what the actual prevalence of this sentiment among deaf people is. I would expect it to be exceedingly low.
I'm not deaf, but I'm legally blind and autistic. Interestingly, I've never once heard of someone take this position with regards to visual impairment. Why is that seemingly so universally agreed upon to be a "real" disability, and things like autism and deafness aren't?
What is particularly striking to me is bundling of two factors here:
- Loss of hearing
- Identity built around loss of hearing
To me these two are distinct. I don't value people based on their disabilities or lack there of. So for me the ability to fix a body's physical deficiency is always a good thing. It makes life better for the person inside the body. These arguments, that I called stupid, conflate both points and assume that seeing lack/loss of hearing as an impediment automatically passes judgement on people who suffer from it.I'd also point out that creating an identity around a feature of one's body is a poor man's substitute for loving yourself. No wonder that people who do that get so defensive. Everything becomes a personal attack to them. While it's understandable, it doesn't make it any smarter, wiser, or functional.
A thing that's striking if you spend any time around deaf people is that at the very top there are exactly two distinct branches of human language: vocal and sign. Once this settles into your understanding the implications are profound and it makes it impossible to dismiss as merely failing to love themselves. (??)
Sign language is exactly as rich a linguistic and cultural tradition as all vocal languages combined, it is an equal branch of human expression & life. It's not the hearing or deafness exactly, it's the experience of being one of the participants in and caretakers of this tiny but vibrant and important domain of humanity.
Sign language can be learnt by hearing people, but spoken languages cannot be learnt by deaf people.
More crucially: Cultures do not have a right to exist. If my culture thriving is straightforwardly at odds with society's physical wellbeing, I need to change and adapt, not society.
Where did I say that:
- Sign language is not rich in importance and tradition
- Sign language is not an equal branch of expression and life
- Sign language, and body language, are not important and have no profound implications
Please, tell me where did I say any of those things.> makes it impossible to dismiss as merely failing to love themselves. (??)
I would encourage you to practice reading with comprehension. I said that building identities around features of one's body is a poor man's substitute of self love. If you don't understand what that means and how it differs from "dismissing deaf people's language as their failure to love themselves", let me know, I will try to explain.
> It's not the hearing or deafness exactly, it's the experience of being one of the participants in and caretakers of this tiny but vibrant and important domain of humanity.
Great, at what point did I suggest that any of that is unimportant, prohibited, unworthy of continuing etc. etc.?
I called this statement stupid:
These kind of genetic therapies seem to reinforce this idea of deafness being a problem in need of eradication
It is a problem and there is a need to solve it. Simply because a healthy person can hear. If we can help restore hearing, how could that be controversial? I don't understand. Btw. using the word "eradication" is already a strong sign of emotional imbalance of the speaker and that the only solution for disabled people to fully assimilate into society is through a medical intervention
If you read the article, noone said anything about this medical procedure being "the only solution (...) to fully assimilate into society". In other words, the person who said this is unhinged.There, that's what I said and meant.
The factor you are missing in the middle is “language and culture developed specifically around this loss of hearing”. The identity isn’t built around lack of hearing, it’s built around a society that will be literally destroyed if the specific feature that mandates membership is eradicated.
As an analogy, how would you feel about a new mandate that all babies learn English as a first language?
I know several hearing people who are part of the deaf culture. They grew up with deaf parents or otherwise interact with that culture to join. I'm sure their kids will not be. Just like my grandpa was part of a German speaking culture that my dad never joined.
> As an analogy, how would you feel about a new mandate that all babies learn English as a first language?
I think it would be wonderful in its effects (I am not a native English speaker), but I don't like the "mandate" part.
As for the other point you are making - the language and culture were developed to work around physical issue of not hearing. Those who have learned the language can continue to use it after regaining hearing. I don't see why those who can hear couldn't learn it if they wanted to (e.g. to communicate with someone who decides to not pursue treatment for whatever reason). I also don't see why preserving something, that solves a problem that now has a better solution, is so important.
It’s not a new idea, you can go learn all about preserving languages and cultures and why and how people care. But even if you don’t do that you can stop giving ignorant takes like “they just have their identity wrapped up in their disability” and say “concern about preserving languages and cultures seems stupid”.
You are now putting words in my mouth. I said the quoted text was stupid. The stupid part was the statement, that framing deafness as a problem to solve is somehow hurtful and wrong.
Since you are very eager to police what I can or cannot do, let me return the favor: you can stop projecting beliefs you are angry about on other people and you can stop fighting those people over those projected beliefs.
In 200 years we'll be having the same debate about whether missing the gene for the Human-Machine Interface Organ is a problem in need of eradication.
It’s not, and if you don’t understand this, you’ve never been around the deaf community before. My college language agreement was filled through sign language. Learning about the deaf community was fascinating.
To some in the Deaf community, being Deaf is like skin color or hair color or height or left handedness; a normal variation of humanity with its own culture. "Fixing" reads as genocide to them, and it's not entirely unwarranted.
Okay. for that to be a reasonable take: curing deafness must then destroy culture.
would that ACTUALLY happen, though? I challenge that assumption.
Take this hypothetical scenario: magic... magically all deafness is gone, suddenly and instantly. Would this destroy friendships? Would this erode personal relationships? Would this destroy (the very useful invention of) sign language? Would this destroy books or media? Would this devastate financially members of this community? would this kill anyone?
Well, besides the secondary effects of suddenly hearing, potentially leading to accidents. Do you actually think any of the above would happen?
I don't actually see anything like that happening. This is conservatism dressed up wearing a minority's hat. This is staunch resistance to change because of fear of lacking the familiar experience using a gross comparison to prevent reasonable analysis.
But I also believe in personal choice. Mandating conversion is not a power I want to give the government in any capacity. I just do not see the 'genocide' argument.
This is an example (like Christianity) about how horrible ideas attach themselves to identity to prevent their excision from their host. If you don't think Christianity is a good idea: suddenly it's a personal affront to them. If you don't think being deaf is an advantage or neutral: suddenly it's a personal affront to them. Be wary of anything attaching itself like this to your identity: you usually get infected when you are too young to have defenses.
This is just the tip of the iceberg. If we are able to cure genetic disease, it's going to save a lot of people's life.
Often times these are rare disease, even though there are some like type 1 diabete, and so they have very little research on them but the gene responsible for the disease are known.
There is a short video on this by Dr. Ben Miles that explains this really succinctly and with visuals.
https://youtube.com/shorts/O8HZw4AbttQ?si=QSCjyDGEIr3Ll-5h
My short interpretation is that you basically inject a virus to mutate cells permanently and it regrows missing hair that takes sound waves and turns them into electrical impulses in the brain.
Absolutely amazing!
Does anyone here know if this would potentially help people with adult SNHL (not genetic)? i.e. for folks who got SNHL when they were >= 25 years old?
Thank you.
Same question as mine came on in mid 40s, though have never been genetically tested
That's great! Kennedy's team is changing the world and how we see treatments. No more lifetime suppression of symptoms, more cures!
You're right, but because nuance isn't allowed on HN, you'll be down voted.
Primarily the focus has shifted to faster approvals with evidence for new methods and drugs coming down to one high quality trial, and removed stipulations for randomized control trials for ultra rare diseases.
> the focus has shifted to faster approvals with evidence for new methods and drugs coming down to one high quality trial, and removed stipulations for randomized control trials for ultra rare diseases
Could you share how RFK's policies helped bring this to market faster? (Not challenging you, by the way. Just need help connecting the dots.)
Seems like the CNPV used is a Trump admin project though no idea how much RFK is involved. If they Warp Speed All The Things that’s a pretty good idea.
> You're right, but because nuance isn't allowed on HN, you'll be down voted.
Nuance is fine. There's not much nuance in the parent post.
Actually I am unaware about what FDA policy changes helped this, can you elaborate ?
It’s in the OP:
> Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner's National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program
That appears to be a late 2025 program based on the dates on the FDA page which would make it a Trump administration policy change. I don’t have any real knowledge here though. I’m just a guy clicking links. But if this is a policy change that’s exciting for approval of the other gene therapies for related conditions once the technology is solved.
0: https://www.fda.gov/industry/commissioners-national-priority...
I do agree that FDA was quite risk averse before especially in areas where the risk - reward would be better for terminally ill or other patients. Having said that this is a different kind of risk reward treatment where the patient might themselves like to try it even if the risks were elevated compared to other post Phase 3 solutions.
AFAIK critics pointed this to be a form of regulatory capture as well.
There has been a string of massive changes under the new admin.
Latest in this area: https://www.fda.gov/news-events/press-announcements/fda-laun...
“President Trump promised to accelerate cures for American families — and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr. “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.”
“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary, MD, MPH. “It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases.”
Now, you can be caught in the fun partisan civil war on emotions or simply look at outcomes. Good work being done at the FDA, just fact.
What actual regulatory changes have they made though?
Here: https://www.fda.gov/news-events/fda-newsroom/press-announcem...
Good faith, bad faith ... does not matter. Decisions and changes are public record. Read for yourself.
> or simply look at outcomes. Good work being done at the FDA, just fact.
A good outcome doesn't mean there was good work being done. You are conflating the two. You can make bad decisions and still have a good outcome.
I'm happy that this looks like a good outcome, but the current FDA is doing terrible work.
You're presumably referring to the ideologically driven cuts at the NIH and a complete misunderstanding of the efficacy-effectiveness gap in macro-level Research outcomes at the FDA? The ones that have led many prominent Professors of Medicine like Celine Gounder to conclude that “The current administration is waging a war on science.”?
RFKs deleterious impact on scientific research and its funding is well documented in the context of the NIH. 2025's Bethesda Declaration ably details the culture of 'fear and suppression' present under RFK, and the $9.5bn in grants and $2.5bn in contracts he had cut, impacting over 2,000 projects. It concludes with a chilling warning regarding plans to cut up to 40% of NIH's $48 billion budget in the future.
https://en.wikipedia.org/wiki/Bethesda_Declaration
At an FDA level, the same strategy was clearly evident last August when Trump fired CDC Director Monarez after clashing heads with RFK over vaccine policies barely a month into her role. Kennedy had demanded she fire career agency officials and commit to backing his own advisers. Four high-ranking officials resigned in support with Monarez.
In a similar vein, RFK then performed a clean sweep of the legacy 17 person vaccine panel in favour of his handpicked eight person vaccine panel – half of whom share ideologue Kennedy’s famous distrust of vaccines. Democrats on the Senate Health Committee summarised it blunty in an open-letter to RFK: “By removing all 17 of ACIP’s members and replacing them with eight individuals handpicked to advance your anti-vaccine agenda, you have put decades of non-partisan, science-backed work – and, as a result, Americans’ lives – at risk."
Moderna CEO Stephane Bancel subsequently said the company would not invest in new phase 3 infectious disease vaccine trials due to growing opposition from U.S. officials to immunizations.
This was then further compounded when the FDA RTF'd Moderna's new Flu Vaccine on spurious grounds in February, with the Alliance for mRNA Medicines calling the decision “unprecedented,” claiming the FDA was in “disarray,” and warned of a “threat to public health.”
Even last month a federal judge concluded RFKs actions re: the panel were not lawful, and that earlier votes by the panel to downgrade recommendations for hepatitis B vaccines for newborns and COVID-19 shots were invalid, blocking the Trump administration’s much publicised overhaul of the childhood vaccine schedule.
The only partisan stance at the moment would be not acknowledging the systemic dismantling of these scientific safeguards and institutions to the detriment of the American population as a whole.
Partisan, to the core.
How about https://www.fda.gov/news-events/press-announcements/fda-elim... ? Game changer for RWE. Not Science?
Or this one: https://www.fda.gov/news-events/press-announcements/fda-acce... ? A call for more research on certain compounds. Not Science?
I know the MSNOW daily hate machine is fun, so engaging and gives life meaning. But laying off the emo crack for a while would open your mind beyond partisan viewpoints.
We can thank RFK for anti-vaccine leadership leading to changes to the childhood vaccine schedule which make preventable diseases harder to stop. You can celebrate more cures all you want, but as most people know, prevention is the best medicine, and the guy is lukewarm on one of our best defenses when it comes to keeping kids safe (vaccines).
And, let's not forget that RFK said: “every Black kid is now, just as a standard, put on adderall, [selective serotonin reuptake inhibitors], benzos, which are known to induce violence. And those kids are going to have a chance to go somewhere and get re-parented, to live in a community where there’ll be no cell phones, no screens, you’ll actually have to talk to people."
So yeah, according to RFK, every black kid in America deserves "re-parenting". He should resign today.
Hopefully they will soon find one for noise induced hearing loss as well.
I'm pretty ignorant in this field.
Does gene therapy treatment correct the issue just in the individual, or does it also correct the genes inherited by the individual's children?
This particular treatment only applies inside the ear, afaik, but it's theoretically possible to apply gene therapies to rewrite more cells, for more systemic diseases.
That might also suffice for sperm, which are constantly being created anew, but I'm not sure that would work for eggs, which are largely formed in gestation. But there might be an egg-specific variant treatment.
I read “Hair Loss” and got excited.
How is it priced? Did they use the sickle cell cure pricing model? $5 less than a cochlear implant?
I was curious too so I googled and CNBC says that it’s free because it’s part of a Trump deal that exempts them from tariffs for 3 years and through which they’ll follow Most Favoured Nation pricing for the US
> The biotech company will also offer the first hearing-loss gene therapy for free to eligible U.S. patients following regulatory approval of the product earlier Thursday.
https://www.cnbc.com/2026/04/23/regeneron-inks-drug-pricing-...
This is not linked to MFN pricing, which only impact Praulant…(PCSK9 for hypercholesterolemia)
This is good news, but I still have concerns.
Otoferlin [1] uses calcium as a cofactor. These mutations happen for a reason. The enzyme is not only located in the ear, but also in the brain and bone marrow [2].
Will there be repercussions if the virus leaves the local area when the therapy is injected?
These OTOF mutation have their highest expression in the Turkish population. Many people with other variations of this gene only experience deafness when they have a fever[3]. So in my opinion, I would like to see ten year outcomes before celebrating.
[1] https://www.uniprot.org/uniprotkb/Q9HC10/entry
[2] https://www.proteinatlas.org/ENSG00000115155-OTOF/tissue
[3] https://www.frontiersin.org/journals/cell-and-developmental-...
The Regeneron press release mentions that the therapy includes a hair cell specific regulator for the gene:
https://investor.regeneron.com/news-releases/news-release-de...
That of course doesn't rule out problems, but the treatment doesn't naively turn on the protein production either.
Patients with fever induced hearing loss regardless of OTOF mutations were specifically excluded (see inclusion criteria on clinical trials.gov for NCT05788536).
With regard to your point about shedding or systemic exposure. If non functional copies of OTOF in the other tissues expressing the gene were to be replaced by functional copies, what is the concern? How would that negatively impact patients? Doesn’t seem like this would/should be a concern.
Also, mutations don’t have to be teleologically beneficial to occur and persist. They can persist because they are not fatal nor do they impair reproductive competence.
There are a lot of risks with retroviral genetic therapy. However, there are a lot of upsides. I think what we need most, is to gain as much knowledge as possible, to ensure we can treat anything untoward as a result.
In terms of 'leaving the local area', there was a recent treatment intended to be done on one eye first, just in case it did not go as planned. It spread to the other eye:
https://www.cam.ac.uk/research/news/gene-therapy-injection-i...
Viral vector DNA was detected in the anterior segment, retina and optic nerve of the untreated eye. The unexpected visual improvement observed in the untreated eyes could therefore reflect the interocular diffusion of rAAV2/2-ND4. Further investigations are needed to confirm these findings and whether other mechanisms are contributing to this bilateral improvement.
Seeing as the eye was directly injected, it's unclear how it spread. Blood, likely.
Yes, some of the vector was likely cleared from the AH and exposed the contra lateral eye
Good to have concerns, but if I was deaf I'd weigh them based on how much I want to hear and discuss the risks with my doctor.
Personally I think I would want to hear once in my life, even if it meant a potentially shorter life.
I'm glad the Trump admin prioritized this.
This was not a political decision. These kind of accelerated paths have been available for years for a wide variety of therapies.