Gene therapy can partly restore sight in blind people, researchers reveal Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light The Guardian - UK · Oct 7
Huntington's Gene Therapy Still Appears to Slow Daily Function Decline at Four Years, but Main Score Misses Significance Four-year AMT-130 data show a lasting daily function signal, but the main Huntington's score lost significance. What families should know. Medical Daily · Oct 1
Gene therapy: The breakthrough that inspired Spider-Man | Euronews Tech Talks Although an inspiration for science fiction movies, gene therapy is real. Here is how it works and what could be improved about it. Euronews · Sep 30
FDA Clears First Gene Therapy for Sanfilippo Syndrome Type A with Shipments to Treatment Centers Within 60 Days The FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome type A. What families should know about access, cost and safety checks. Medical Daily · Sep 18
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 · Sep 3
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 · Aug 25
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 · Aug 21
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 · Aug 12
11-year-old becomes first UK patient to receive gene therapy for rare condition IN FULL The Independent UK · Jul 24
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 · Jul 23
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 · Jul 23
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 · Jul 21
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 · Jul 15
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 · Jul 13
Medical Daily · Jul 10 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 · Jul 8 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 · Jul 6 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.
The Guardian - US · Jun 24 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’
Medical Daily · Jun 19 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 · Jun 11 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 · Jun 10 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 · May 31 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.
MarketBeat · May 26 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 · May 17 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 · Apr 23 '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.