The FDA has approved the first gene therapy for inherited deafness (Otarminy by Regeneron), which restored hearing in 16 out of 20 children in trials, with 5 achieving near-normal hearing, and the company is providing it free as a gift to humanity. This same gene therapy approach is being applied to treat inherited blindness, sickle cell disease, muscular dystrophy, and hemophilia. Additionally, Altos Labs has demonstrated that cellular rejuvenation can double the remaining lifespan of elderly mice in a single treatment, and Life Biosciences has received FDA approval to test this approach in humans for age-related vision loss. New blood tests can now measure the biological age of individual organs, enabling early intervention before disease manifests.
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Added:Three weeks ago, the FDA approved the first ever gene therapy for inherited deafness. This is not a drill. It's approved. Babies who would have grown up profoundly deaf can now hear. I'm Brit, a serial entrepreneur and investor at Offline Ventures. Health is one of my biggest investing areas, and right now, the pace of biotech breakthroughs is making me genuinely emotional. Let me tell you why. That deafness therapy is called Otarminy, made by Regeneron. It targets a mutation in the gene that causes deafness. In trials, 16 out of 20 kids had a measurable hearing improvement within 5 months. Five of them had hearing restored to essentially normal. Here's the part that broke me.
Gene therapies like this usually cost millions of dollars per patient, but Regeneron is providing this for free.
The CEO literally said they wanted to make an example of biotech delivering a gift to humanity. Amazing. And here's the wild part. This is just the beginning. The same playbook, find the broken gene, deliver a working copy, change a life, is being run right now for inherited blindness, sickle cell disease, muscular dystrophy, hemophilia, and dozens more conditions. These things that used to be a life sentence are getting cured, like actually cured. Then there's the longevity side of things.
I'm also fascinated by this. There's a startup called Altos Labs, launched in 2022 with $3 billion. Yes, billion with a B, to work on cellular rejuvenation.
So, partially reprogramming your cells back to their younger state. Crazy. In a landmark mouse study, they gave the gene therapy to elderly mice. So, basically the equivalent of a 75-year-old human, and those mice lived twice as long as the untreated ones from that point forward, in a single shot. Doubled the remaining lifespan. Isn't that wild? And this isn't just mice anymore. In January, a competitor called Life Biosciences got the FDA green light to test the same approach in humans for the first time ever. Their drug ER-100 targets age-related vision loss, specifically glaucoma and a condition where people literally go to to with normal vision and wake up blind in one eye. The trial's happening right now and initial data could come in the next year. On top of all of this, you can now take a blood test that tells you the biological age of your individual organs. Your heart's age, your liver's age, your immune system's age. Imagine knowing your kidneys are aging 10 years faster than the rest of you and being able to intervene before it shows up as disease. This is where it's going. 10 years ago, gene editing and cellular programming were lab curiosities. Today, there's a 2-year-old named Miles who was born pro family deaf and is now hearing his friends talk for the first time. And he's the first of many. If you're building in longevity, gene therapy, or precision health, I'd love to hear from you. And if you've done any biological testing on yourself, drop what you found in the comments. I'm clearly obsessed with this stuff.
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