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EXPLAINED: 2 | Vintage Vibes

Experimental gene therapy successfully treats dozens of children with rare fatal disorder

'These results are what we hoped for when we first began developing this approach,' one researcher said.

Euronews

FDA Acceptance Of Rocket Pharmaceuticals' Lead Gene Therapy Application Reestablishes Momentum For Stock

On Tuesday, the U.S. Food and Drug Administration (FDA) accepted Rocket Pharmaceuticals, Inc.’s (NASDAQ:RCKT) resubmission of the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).

Benzinga

Regeneron's Gene Therapy Shows Significant Gains In Genetic Hearing Loss

On Sunday, Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) unveiled updated data from its investigational gene therapy DB-OTO for profound genetic hearing loss caused by variants of the otoferlin (OTOF) gene.

Benzinga

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read the most diverse news in one place.

A new treatment for Huntington’s disease is genuinely promising – but here’s why we still need caution

Imagine knowing in your 20s or 30s that you carry a gene which will cause your mind and body to slowly unravel. Huntington’s disease is inherited, relentless and fatal, and there is no cure. Families live with the certainty of decline stretching across generations.

The Conversation

New hope for Huntington’s families as gene therapy shows remarkable results

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington’s disease. The news has spread quickly through families affected by this condition, who have been desperately waiting for a treatment that can stop or slow down this devastating illness.

The Conversation

Friday briefing: The Huntington’s treatment is a ray of light for victims of a brutal disease

In today’s newsletter: A gene therapy trial has successfully treated Huntington’s for the first time – one of several recent groundbreaking medical advances bringing hope in gloomy times

The Guardian - UK

UniQure Stock's 248% Blastoff Was Just The Start — Ask Wall Street

UniQure NV (NASDAQ: QURE) just staged one of biotech's wildest rides of the year, soaring 248% in a single session after unveiling successful trial results for its experimental Huntington's disease gene therapy. Indeed, the stock reached a 5-year high, marking its best session ever. But if you think the move…

Benzinga

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Doctors say experimental gene therapy seems to slow Huntington’s disease for first time

It is the first time a drug trial has slowed the rare disorder’s progression in an ongoing, significant way, researchers said.

Euronews

This Stock Is Tripling on a Key Gene Therapy Win. Should You Buy the 200%-Plus Surge Now?

Uniqure shares skyrocket as the biotech firm announces a major breakthrough in Huntington’s disease. Here’s why investors should wait before initiating a new position in QURE stock.

Barchart

'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

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

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The Independent UK

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

Benzinga

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.

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LiveScience

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.

Benzinga

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.

The Conversation

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 Guardian - US

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

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.

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