hotspot -WSS

hotspot

author：fashion 2025-09-29 20:51:07 - browse：6163

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： Hepatitis C has a cure — but many Americans still lack access to it
Next article： Depression: FDA OKs Otsuka Pharmaceuticals digital therapeutic

WSS

Alzheimer's risk factor APOE4 may be a distinct form of the disease

Oral obesity drug from Viking Therapeutics hits key early target

People with mental disorders often marry others with same

Bring on the sabbaticals for nurses

Alzheimer's risk factor APOE4 may be a distinct form of the disease

Overprescribed: High cost isn't America's only drug problem

knowledge

hotspot

This article is exclusive to STAT+ subscribers

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

entertainment

Hollywood unions extend contract negotiations for actors

Hundreds died while taking an arthritis drug, but nobody alerted patients

Peter Hotez and the public health issue of online harassment

Bird flu in dairy cattle: New measures to control spread to humans

Walensky prepares to leave CDC — and Congress — behind

Alzheimer's risk factor APOE4 may be a distinct form of the disease

In Memoriam: Notable people who died in 2023

Medicare Advantage insurers pressure Biden for bigger payments

Hepatitis C has a cure — but many Americans still lack access to it

Covid silver lining: Unprecedented look at human immune system

knowledge

Pancreatic cancer is often deadly, but new discoveries are raising hope

Biotech investment managers search for lucrative opportunities

Steward Health Care expansion into Malta flopped

Covid silver lining: Unprecedented look at human immune system

Study: Black kids more likely to die after surgery than white kids

Medicare Advantage 2025 payments will dip, Biden administration says

Wikipedia

explore

hotspot

knowledge