explore -WSS

explore

author：leisure time 2025-09-06 01:57:56 - browse：3941

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： Medicare details structure of new drug price negotiation program
Next article： FDA's naming rule on biosimilars has undermined competition

WSS

Senate panel clears PBM, hopsital reforms

Covid vaccine financial winners and losers, from Pfizer to Novavax

Roche subsidiary Foundation Medicine opens new headquarters

Primary care physicians play key roles in Alzheimer's management

There's no autism epidemic. It's an autism diagnosis epidemic

The Sacklers, horror, and ‘The Fall of the House of Usher’

hotspot

explore

This article is exclusive to STAT+ subscribers

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

entertainment

In age of alternative facts, a scholarly course on calling out crap

Excited delirium, Elijah McClain, and emergency physicians

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

STAT Executive Editor Rick Berke on STAT's 8th birthday

Jerome Adams on the need for new antivirals for Covid

10 million people disenrolled from Medicaid over past six months

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Donor acceptance rates hint at racial, gender bias in heart transplants: study

As demand for Covid shots wanes, Moderna seeks its next success

Victims of gun violence feel impacts long after bullet wound

focus

More doctors should go into startup world

Steward Health Care has deal to sell doctor network to UnitedHealth

Readout Newsletter: Blackstone Life Sciences, Merck, Bluebird Bio

Patient advocates debate FDA's accelerated approval process

Removing probiotics from the NICU could threaten preemies' health

Washington mulls new rules to avert another Change Healthcare

focus

hotspot

focus

Wikipedia