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The Latest Breaking News from Gene Therapy

'Groundbreaking' gene therapy is first treatment for Huntington's disease to slow the condition

Results from a three-year trial suggest an experimental gene therapy for Huntington's disease can slow the progression of the deadly condition by 75%.

LiveScience

First Successful Gene Therapy for Huntington's Disease Slows Disease Progression by 75%

In a landmark clinical trial, uniQure's gene therapy AMT-130 slowed Huntington's disease progression by 75% over 3 years. The one-time treatment may mark the first disease-modifying therapy for this incurable neurodegenerative condition.

Medical Daily

Huntington’s disease treated successfully for first time in UK gene therapy trial

Surgical procedure to treat devastating illness slowed progress of disease by 75% in patients after three years

The Guardian - UK

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Science history: A tragic gene therapy death that stalled the field for a decade — Sept. 17, 1999

Sept. 17, 1999: Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation and caution around gene therapy, which ultimately stalled the field for years.

LiveScience

FDA Expands Krystal Biotech Vyjuvek's Label Allowing Newborns Access To Gene Therapy For Blistering Skin

The U.S. Food and Drug Administration (FDA) on Monday approved Krystal Biotech, Inc.’s (NASDAQ:KRYS) label update for Vyjuvek (beremagene geperpavec-svdt).

Benzinga

Mom unable to get treatment for one of two boys with rare genetic disorder gets life-changing update: ‘Don’t give up’

Exclusive: Hunter Small, 10, was initially denied gene therapy for Duchenne muscular dystrophy after his brother Noah, 9, received treatment

The Independent UK

Two brothers have the same genetic disorder. Their insurance only covered life-altering treatment for one of them

Exclusive: Alison Joseph's sons Noah and Hunter both have Duchenne muscular dystrophy. She tells Rhian Lubin the heartbreaking reality of watching one thrive while the other suffers

The Independent UK

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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

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

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International Business Times UK

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.

Benzinga

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.

The Independent UK

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

Euronews

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.

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Benzinga

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.

The Independent UK

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

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.

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