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THE TRUTH ABOUT: Gene therapy - The Untold Story

uniQure Files for US Approval of Its Huntington's Gene Therapy and Asks Regulators for Faster Review

uniQure filed for FDA approval of AMT-130, a one-time Huntington's gene therapy, and requested priority review. Here is what it means.

Medical Daily

Hunter Syndrome Gene Therapy Halted by FDA After Spine Scans Show Small Masses in Five Children

FDA placed REGENXBIO's RGX-121 on clinical hold after spine MRIs showed nodules or cysts in five Hunter syndrome trial participants.

Medical Daily

Gene Therapy for Glycogen Storage Disease Type Ia Wins Accelerated Approval Priced at 2.7 Million Dollars

FDA granted accelerated approval to Genglycos for glycogen storage disease type Ia. It reduces cornstarch use, and the long-term benefit is unproven.

Medical Daily

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Cardinal Health Earnings: Can Perfection Get Priced In Twice?

Cardinal Health stock hit a new all-time high after mixed earnings, with analyst price target hikes, gene therapy logistics wins and a 29-year dividend growth streak supporting the uptrend.

MarketBeat

11-year-old becomes first UK patient to receive gene therapy for rare condition

IN FULL

The Independent UK

Girl, 11, becomes first UK patient to have ‘groundbreaking’ gene therapy to save her sight

Catherine L’Estrange, from North Acton in London, is hopeful the surgery will allow her to continue one of her favourite hobbies – reading books

The Independent UK

11-year-old girl is first UK patient to have gene therapy to halt sight loss

Catherine L’Estrange was diagnosed with a rare genetic condition as a baby and hopes the treatment will allow her to keep reading books.

Evening Standard

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FDA Expands CRISPR Gene Therapy Casgevy to Children as Young as 2 with Sickle Cell Disease

FDA approved Casgevy CRISPR gene therapy for children as young as 2 with sickle cell disease on July 1, 2026. Here's what families need to know about this milestone.

Medical Daily

FDA drug approval affects healthcare around the world, but political shortcuts could hurt the agency’s international reputation

At the beginning of July 2026, the U.S. Food and Drug Administration approved the first gene therapy for young children with sickle cell disease.

The Conversation

India developing indigenous solutions to global health challenges: Rajnath Singh

India is setting new benchmarks in medical research and indigenous solutions. Gene therapy and nuclear medicine are key areas of advancement for global health. The nation's health system is becoming more self-reliant and affordable for citizens. Indigenous manufacturing of medical equipment has gained momentum through new schemes. Uttar Pradesh has…

The Economic Times

Harvard Built a Silicon Chip That Writes DNA Using Electricity and Water: What It Could Mean for Medicine

Harvard researchers created a silicon chip that writes 64 DNA sequences in parallel using only electricity and water-based enzymes, a potential step toward cheaper gene therapies.

Medical Daily

Medicaid Will Cover Sickle Cell Gene Therapy, but Only When It Actually Works for the Patient

CMS's CGT Access Model now covers Casgevy and Lyfgenia for sickle cell through 33 state Medicaid programs under outcomes-based contracts. Here's what patients need to know.

Medical Daily

A 23-Year-Old Louisiana Man Became the First Person in the Gulf South to Be Functionally Cured of Sickle Cell Disease

Daniel Cressy, 23, is the first person in the Gulf South functionally cured of sickle cell disease using Casgevy CRISPR gene therapy at Manning Family Children's Hospital in New Orleans.

Medical Daily

Louisiana man becomes first in region functionally cured of sickle cell disease

Daniel Cressy, 23, says path leading to completion of curative gene therapy is his ‘greatest blessing’

The Guardian - US

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Medical Daily

Huntington's Disease Gene Therapy AMT-130 Is on Track for FDA Approval After uniQure Reaches Regulatory Agreement

uniQure announced June 17, 2026: FDA agreed that 3-year AMT-130 Phase I/II data supports an accelerated approval BLA for Huntington's disease. BLA submission planned Q3 2026.

AAP

Lifespan Portfolio Company Rejuvenate Bio Closes $6 Million Financing and R&D Collaboration with Merck Animal Health

NORWALK, Conn., June 11, 2026 /PRNewswire/ -- Lifespan Vision Ventures (LVV), an investment firm focused on therapeutics that improve human healthspan, congratulates its portfolio company Rejuvenate Bio on the successful close of a $6 million financing round and the announcement of a strategic research and development collaboration with Merck Animal…

Euronews

Is it possible to rejuvenate damaged human cells? US biotech firm tests new gene therapy

A US biotechnology company has tested a gene therapy designed to reverse ageing in humans for the first time.

International Business Times UK

Putin Bills Russians $26 Billion For Constant Organ Transplants & New Drug To Survive

Russia invests £22 billion in anti-ageing technologies to extend political leadership lifespan. The programme includes organ cultivation and gene therapy, aiming to secure a permanent political dynasty.

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MarketBeat

Opus Genetics Touts Retinal Gene Therapy Pipeline, September BEST1 Data Catalyst

Opus Genetics (NASDAQ:IRD) is advancing a portfolio of gene therapies for inherited retinal diseases, with two programs already in the clinic and three more expected to enter clinical testing within the next 12 months, President Dr. Ben Yerxa said during a fireside chat at RBC Capital Markets’ 2026

MarketBeat

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

LiveScience

'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.

The Independent UK

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

IN FULL

The Independent UK

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

Evening Standard

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.

LiveScience

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.

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