hotspot -WSS

hotspot

author：focus 2025-10-01 00:06:57 - browse：69

A syringe injects into a segment of DNA — biotech coverage from STAT — Adobe

Two congenitally deaf children can hear for the first time after being treated with gene therapy, according to data presented at a conference Wednesday.

The results are “jaw-droppingly good, just shocking how good. It exceeded the wildest expectations of anybody who started this work,” said Larry Lustig, an otolaryngologist at Columbia University and an investigator on the study.

The new data come from a larger study by Regeneron Pharmaceuticals that will treat nearly two dozen children with a specific type of congenital hearing loss with gene therapy. The clinical trial is meant to measure safety and efficacy of DB-OTO, a gene therapy drug Regeneron is developing to treat children who have hearing loss due to changes in the otoferlin gene. Several teams are targeting this rare form of congenital deafness that affects 1% to 8% of deaf people globally, including a team with Akouos, an Eli Lilly subsidiary, which successfully treated an 11-year-old boy with a similar gene therapy. Lustig calls this the “lowest of the low-hanging fruit” for gene therapy deafness treatments.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Tori Bowie and the unacceptable rates of Black maternal death
Next article： 'Reaching a detente': STAT's Helen Branswell on covering Covid

WSS

Gilgo Beach murders: What questions remain following Rex Heuermann's arrest?

Ebola public health emergency is over, WHO declares

Trump got a 'target letter.' What is one and what does it mean?

The surgeon had a dilemma only a Nazi medical text could resolve

Why the Crimean Bridge is key to Russia's war in Ukraine

New warning issued for rebooking air travel after delays, cancellations

Wikipedia

hotspot

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

focus

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

What to make of Gilead’s Trodelvy troubles and Elevation Oncology’s rise

Drug repurposing or repositioning? The language matters

Resistance to cancer drugs: A new genetic explanation

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Medical experts are missing from AI regulatory conversations

What does generative AI mean for health care? We asked experts

Think you might have Covid

After affirmative action ruling, medical educators look to 'holistic review'

Readout Newsletter: BridgeBio, Madrigal, and Silicon Valley Bank

focus

Test for long Covid? Researchers find new clue in blood samples

Researchers separate signal from noise in heart wearable data

Why doctors need education on FDA device approval process

Medicare Advantage today, explained in one chart

Test for long Covid? Researchers find new clue in blood samples

Gilead’s Trodelvy fails in lung cancer, raises questions on ADCs

Wikipedia

focus

hotspot

focus