Get all your news in one place.

100’s of premium titles.
One app.

Get all your news in one place.

100’s of premium titles. One news app.

The Latest Breaking News from Gene Therapy

Regenxbio Unveils 12-Month Data For Gene Therapy Soon After FDA Delays Review Date

On Friday, REGENXBIO Inc. (NASDAQ:RGNX) shared new data from the Phase 1/2/3 CAMPSIITE trial of clemidsogene lanparvovec (RGX-121) for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025.

Benzinga

uniQure's Investigational Gene Therapy Shows Promising Efficacy With Manageable Safety Risks For Fabry Disease

uniQure N.V. (NASDAQ:QURE) on Friday released initial safety and exploratory efficacy data from the first cohort of its Phase 1/2a trial of AMT-191, an investigational gene therapy for Fabry disease.

Benzinga

Gene that human ancestors lost millions of years ago could help treat gout

Researchers used evolutionary genetics and CRISPR gene editing tech to develop an innovative treatment for gout. The approach has yet to be tested in humans.

LiveScience

From analysis to the latest developments in health,
read the most diverse news in one place.

FDA Lifts Rocket Pharmaceuticals Study Hold For Rare Disease Gene Therapy

Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) stock is trading higher on Wednesday after the U.S. Food and Drug Administration (FDA) lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease.

Benzinga

FDA Extends Decision Date On Regenxbio's Gene Therapy Into Next Year

The U.S. Food and Drug Administration (FDA) on Monday extended its review timeline for REGENXBIO Inc.’s (NASDAQ:RGNX) Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.

Benzinga

Gene therapy can be less effective in women – and my research in mice brings us one step closer to understanding why

Gene therapies hold immense promise for treating sight loss. These therapies use a modified, harmless virus to deliver therapeutic genes directly to diseased cells, helping them to function normally again.

The Conversation

Vinay Prasad returns to FDA days after leaving under pressure from Laura Loomer

Prasad left the agency after far-right influencer released edited audio misleadingly suggesting he was anti-Trump

The Guardian - US

Read news from The Economist, FT, and more,
with one subscription

Vinay Prasad vs the FDA: How One Doctor's Policies Collided with Politics and Cost Him His Job

Vinay Prasad resigns from the FDA after backlash over vaccine restrictions and gene therapy decisions. Political pressure cited in sudden departure.

International Business Times UK

What's Going On With Sarepta, Capricor And Other Gene Therapy Stocks On Wednesday?

In May, FDA Commissioner Marty Makary named Vinay Prasad the next director of the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER). Within three months, Prasad resigned.

Benzinga

A single injection for newborns could protect them against HIV for years, study suggests

Globally, an estimated 1.3 million women and girls living with HIV become pregnant every year, according to the World Health Organization

The Independent UK

Experimental treatment restores sight to man with genetic eye disorder in Italy

A patient suffering from a genetic eye disorder in Italy regained his sight thanks to a world-first experimental gene therapy.

Euronews

FDA Lifts Safety Hold On Sarepta's Muscular Dystrophy Gene Therapy—Wall Street Cheers

As gene therapies continue to evolve as a promising frontier in medical treatment, regulatory scrutiny intensifies following safety concerns. Recent developments with Sarepta Therapeutics Inc (NASDAQ:SRPT) highlight the delicate balance between innovation and patient safety in the biopharmaceutical industry.

Benzinga

Sarepta Therapeutics Surges Over 40% In Tuesday Pre-Market: What's Going On?

Stock of Sarepta Therapeutics Inc. (NASDAQ:SRPT) surged 41.05% in Tuesday pre-market following the U.S. Food and Drug Administration’s (FDA) recommendation to lift the voluntary hold on Elevidys, Sarepta’s gene therapy for Duchenne Muscular Dystrophy.

Benzinga

Sarepta will resume gene therapy shipments after FDA review of recent patient death

Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping its gene therapy

The Independent UK

Get all your
news in one place.

100’s of premium titles.
One news app.

Benzinga

FDA Probes Death Of 8-Year-Old Who Received Sarepta's Elevidys

The U.S. Food and Drug Administration (FDA) announced on Friday, after market close, that it is investigating the death of an 8-year-old boy who had received Elevidys, Sarepta Therapeutics Inc.’s (NASDAQ:SRPT) gene therapy for Duchenne muscular dystrophy.

Benzinga

Rocket Refocuses Pipeline As Gene Therapy Sentiment Sours

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a firm focused on developing cardiovascular gene therapy, announced a strategic corporate reorganization to extend its runway.

Benzinga

Sarepta Gets Thumbs Down From Regulators Regarding Elevidys Gene Therapy

On Friday, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

Benzinga

Elevidys Halt Sparks Analyst Downgrades—Sarepta Races To Revise Label

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Monday it will voluntarily and temporarily pause all U.S. shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, effective July 22.

Get all your
news in one place.

100’s of premium titles.
One news app.

The Independent UK

Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators

Benzinga

Sarepta Reverses Course, Pauses Elevidys Shipments Amid FDA Safety Review

Benzinga

Sarepta Faces Regulatory Showdown As Analysts Cut Price Targets

On Friday, the U.S. Food and Drug Administration placed Sarepta Therapeutics Inc.’s (NASDAQ:SRPT) investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD) on a clinical hold.

Benzinga

Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys

The ground shook for Sarepta Therapeutics, Inc. (NASDAQ:SRPT) last week as the U.S. Food and Drug Administration (FDA), alarmed by a third patient death tied to its gene therapy platform, pulled a rare move: requesting a voluntary halt to Elevidys shipments. The company’s response? A resounding “no,” igniting a major…

The Independent UK

Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene therapy

Benzinga

Sarepta's Setback Deepens With Another Gene Therapy Fatality

Sarepta Therapeutics (NASDAQ:SRPT) shares plummeted on Friday following reports of a third patient death linked to its gene therapy pipeline this year, further clouding investor confidence despite the company’s recent efforts to provide clarity on its restructuring and FDA label plans.

Benzinga

Sarepta Therapeutics Stock Plunges Over 30% In Friday Pre-Market: What's Going On

Shares of Sarepta Therapeutics (NASDAQ:SRPT) plunged over 30% in Friday pre-market after a clinical trial participant died following treatment with a gene therapy, marking the third death linked to the company’s experimental treatments.

1 2 3 4 5

People

From analysis to the latest developments in health,
read the most diverse news in one place.

Organisations

the World Health Organization

the Department of Health and Human Services

128 stories

the US Food and Drug Administration

Read news from The Economist, FT, and more,
with one subscription

Topics

Places

Fourteen days free

Download the app

One app. One membership.
100+ trusted global sources.