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The Latest Breaking News from Gene Therapy

Groundbreaking UK gene therapy offers hope after progress of three-year-old

Oliver Chu from California first person to have the one-off treatment for Hunter syndrome

The Guardian - UK

Gene Editing Medicine and Personalized Medicine: Breakthroughs Driving Health Innovation Gene Therapy to Transform Care

Explore how gene editing medicine and personalized medicine future breakthroughs are revolutionizing health innovation gene therapy, offering precise, transformative treatments and medical breakthroughs for complex diseases.

Medical Daily

FDA adds strongest warning to Sarepta gene therapy linked to 2 patient deaths

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year

The Independent UK

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Gene Therapy for Children With Rare 'Bubble Boy Disease' Proves Effective After Long-Term Follow-Up

A new gene therapy for children with the rare "Bubble Boy disease" proved to be effective following the results of long-term follow-ups.

Medical Daily

Eli Lilly Expands Genetic Eye-Disease Focused Pipeline With MeiraGTx Gene Therapy Collaboration

MeiraGTx Holdings plc (NASDAQ:MGTX) stock is trading higher after the company inked a strategic collaboration in the area of ophthalmology with Eli Lilly and Co. (NYSE:LLY).

Benzinga

Eli Lilly Bets Big On Eye Gene Therapy That Could End Repeat Injections

Eli Lilly and Company (NYSE:LLY) and Adverum Biotechnologies, Inc. (NASDAQ:ADVM) on Friday announced that they have entered into a definitive agreement under which Lilly will acquire Adverum Biotechnologies, including its lead product, Ixo-vec.

Benzinga

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

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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

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

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

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Barchart

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.

LiveScience

'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%.

Medical Daily

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.

The Guardian - UK

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

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LiveScience

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.

Benzinga

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).

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.

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.

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