hotspot -WSS

hotspot

author：Wikipedia 2025-10-02 10:38:36 - browse：168

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： Bright Health sells Medicare Advantage business to Molina
Next article： 'Screen and refer' systems are hurting patients

WSS

Correcting Robert F. Kennedy Jr.'s vaccine 'facts'

How a symptom of dementia informs our understanding of creativity

Archived: an online chat discussing Ebola, vaccines, and a new epidemic

Grandmother of French teen shot dead by police officer pleads with rioters to stop the violence

What AAP's gender

'Screen and refer' systems are hurting patients

hotspot

hotspot

This article is exclusive to STAT+ subscribers

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

hotspot

Affirmative action in medical school literally saved lives

Complete sequence of Y chromosome published for first time

New air purifier tech could be key to improving indoor air quality

How a symptom of dementia informs our understanding of creativity

Moonlake's readout produced a cash windfall. Risks remain

What do new weight loss drugs mean for bariatric surgery?

At least 13 dead in Texas as scorching temps continue

How Long Island became the ‘eugenics capital of the world’

Guidelines to prevent youth baseball injuries need more muscle

What affirmative action's end means to a medical student

knowledge

Winners, losers from Medicare’s list of drugs subject to price talks

AstraZeneca lung cancer study comes in below expectations

Archived: an online chat discussing Ebola, vaccines, and a new epidemic

Medicare explains how it will choose drugs for negotiation

'Screen and refer' systems are hurting patients

In science, is the line between industry and academia shifting?

focus

knowledge

entertainment

explore