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The Latest Breaking News from Gene Therapy

4D Molecular Therapeutics Touts Rapid Phase 3 Momentum for Retinal Gene Therapy

4D Molecular Therapeutics (NASDAQ:FDMT) executives said the company is seeing strong clinical trial momentum for its lead retinal gene therapy candidate, 4D-150, as it advances through late-stage development for wet age-related macular degeneration and prepares to begin a Phase 3 trial in diabetic m

MarketBeat

'A landmark moment for the field': FDA approves first-ever gene therapy for inherited deafness

A gene therapy made by Regeneron is the first treatment of its kind approved for genetic hearing loss.

LiveScience

Mother hails gene therapy treatment that restored daughter’s sight as ‘life-changing’

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The Independent UK

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Six-year-old girl has vision restored after first-of-its-kind NHS eye gene therapy

Saffie Sandford was diagnosed with the rare condition Leber’s Congenital Amaurosis (LCA) that affects vision in low light

The Independent UK

Six-year-old girl has sight restored thanks to life-changing eye gene therapy

Saffie Sandford had a first of its kind treatment for one of the genetic causes of her condition.

Evening Standard

Gene therapy improves hearing in 90% of patients with inherited deafness in largest trial of its kind

A new gene therapy tested in China has improved the hearing of 38 people who were born deaf due to mutations in a gene called OTOF.

LiveScience

‘Oscar of science’ awarded to team behind gene therapy that restores lost vision

Married couple Jean Bennett and Albert Maguire developed Luxturna, which helped a patient see their child’s face for the first time

The Guardian - UK

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$3 million prize goes to duo whose research led to first sickle cell CRISPR therapy

Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a functional cure for the deadly blood disorders sickle cell disease and beta thalassemia.

LiveScience

Hearing Loss Breakthrough: The New Gene Therapy That Could Restore Hearing in Older Adults

Imagine being able to hear clearly again—without hearing aids, without implants, and without constant adjustments. That’s the promise behind a new wave of gene therapy...

Saving Advice

The Rise of Longevity Medicine: How Anti-Aging Clinics Promise Longer, Healthier Lives

Longevity medicine and anti aging clinics promise longer, healthier lives with biomarker testing, gene therapy, and personalized treatments.

Medical Daily

Forbes’ 250: Who are the 11 Indian Americans recently named America’s Greatest Innovators?

Eleven Indian Americans have been celebrated on Forbes' America's Greatest Innovators list, highlighting their transformative contributions across technology, biotech and infrastructure. These leaders, from AI pioneers to gene therapy developers, underscore the vital role of immigrant talent in shaping America's technological and economic future, inspiring a new generation of innovators.

The Times of India

UAE launches pioneering gene therapy to treat autism and rare brain disorders in children

The UAE has launched two gene therapy projects targeting the root causes of autism and rare brain disorders, marking a significant shift from symptom management to biological repair. Collaborating with global experts, these initiatives aim to enhance treatment effectiveness and safety for children with conditions like BPAN, paving the way…

The Times of India

As billionaires chase immortality, this startup cofounded by a Harvard genetics professor gets FDA approval for the first partial de-aging human trial

Life Biosciences will test its gene therapy designed to partially reset a cell’s age and potentially reverse vision loss in patients with glaucoma and NAION.

Fortune

Retired HGV driver undergoes heart bypass using gene therapy to reduce risks

The procedure aims to extend the lifespan of blood vessels used during the graft process in the surgery.

Evening Standard

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LiveScience

From gene therapy breakthroughs to preventable disease outbreaks: The health trends that will shape 2026

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in 2026.

The Guardian - UK

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

Medical Daily

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.

The Independent UK

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

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Medical Daily

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.

Benzinga

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.

Euronews

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.

Benzinga

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.

The Conversation

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.

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