explore -WSS

explore

author：hotspot 2025-09-27 16:11:40 - browse：56925

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： George Santos makes 1st court appearance after pleading not guilty to 13 counts
Next article： Israel withdraws forces after 2

leisure time

WSS

The strategy behind pharma's lawsuits over drug pricing reform

What is the Wagner Group? The 'brutal' Russian military unit in Ukraine

Man pleads guilty to raping Ohio girl who ended up traveling across state lines for abortion

Search warrant issued in 1996 disappearance of Kristin Smart

1 in 4 Americans hadn't contracted COVID by the end of 2022, CDC estimates

Paxlovid study fails to answer questions over benefit for broader groups

focus

explore

This article is exclusive to STAT+ subscribers

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

focus

Medicare proposal to cover medical devices draws a mixed reaction

#NeedHerScience: shining a light on gender disparities in medicine

Apple is now the first public company to be valued at $3 trillion

Man with guns arrested near Obama home 'attempted to evade' Secret Service: Prosecutors

Walensky prepares to leave CDC — and Congress — behind

3 hurdles to bringing medical devices to the U.S. market

Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

Supreme Court shows surprising restraint in chaotic year of crises: ANALYSIS

State Dept. review finds Biden bears some blame for Afghanistan failures

CDC concerned about possible undetected monkeypox spread in U.K.

entertainment

'Insanity': 4th of July mass shootings across US kill 13, injure more than 80

Why are elite athletes different? Take a look at their microbes

Why are elite athletes different? Take a look at their microbes

What is the Wagner Group? The 'brutal' Russian military unit in Ukraine

1 in 4 Americans hadn't contracted COVID by the end of 2022, CDC estimates

Dramatic video released of Russian fighter jets 'harassing' US drones over Syria

leisure time

focus

focus

leisure time