hotspot -WSS

hotspot

author：focus 2025-08-02 19:12:42 - browse：6

portrait of Jennifer Adair -- coverage from STAT — Jennifer Adair develops scalable, low cost gene therapies at the Fred Hutchinson Cancer Center and helped found the Global Gene Therapy Initiative. Fred Hutch News Service

BALTIMORE — Gene and cell therapies have transformed a handful of devastating disorders in the U.S. and Europe, with more treatments on the way. But those technologies have largely failed to reach most of the world.

These lags are ubiquitous in the current drug development system, where new therapies are approved in wealthy countries and then slowly leak out to low- and middle-income countries, many years later. In the case of gene therapy, that means countries most affected by a disease such as sickle cell may wait decades to access a curative therapy.

For the last few years, a handful of researchers in the U.S. and around the world have tried to find ways of ensuring global access to these technologies. In 2020, they formed the Global Gene Therapy Initiative, with an initial goal of setting up a gene therapy clinical trial for a hemoglobinopathy — such as a disease like sickle cell or beta thalassemia — or HIV in two different low- and middle-income countries by 2024.

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： Moonlake's readout produced a cash windfall. Risks remain
Next article： Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

leisure time

WSS

The cancer drug shortage isn’t new — and neither are the solutions

23andMe had bad news about my health. I wish a person had delivered it

United colonoscopy coverage change 'may cost lives,' doctors say

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

Britain’s MI6 chief says his spies are using AI to disrupt flow of weapons to Russia

Biden administration sends mixed signals on US soldier who crossed into North Korea

focus

hotspot

This article is exclusive to STAT+ subscribers

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

explore

Hollywood unions extend contract negotiations for actors

Medicare details structure of new drug price negotiation program

Medicare proposal to cover medical devices draws a mixed reaction

Supreme Court strikes down use of affirmative action

Bright Health sells Medicare Advantage business to Molina

BioMarin wins approval for gene therapy to treat hemophilia A

The cancer drug shortage isn’t new — and neither are the solutions

At least 13 dead in Texas as scorching temps continue

DIEP, the 'gold standard' of breast reconstruction, is under threat

FDA seeking to stop procedures at two unregulated stem cell clinics

knowledge

Flagship, Pfizer team up in deal worth up to $7 billion

Biden administration sends mixed signals on US soldier who crossed into North Korea

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

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

Johnson & Johnson sues to stop Medicare negotiation

Manchin plays down potential 3rd

leisure time

leisure time

focus

focus