Articles by tag: gene-therapy
AAV gene therapy for HoFH: cholesterol reduction by 97.5% in clinical trial
New AAV8 gene therapy NGGT006 for homozygous familial hypercholesterolemia reduced LDL-C by 97.5% in 3 weeks. Effect persists for 52 weeks. Find out who is eligible for treatment.
CRISPR vision restoration: FDA approval for testing
A US startup received FDA approval to test CRISPR therapy for people born blind with Leber congenital amaurosis. Details, risks, and prospects in the article.
Gene therapy for narcolepsy: why Stanford is late
NEJM reports on gene therapy for narcolepsy in mice, but Takeda is already preparing to launch oveporexone. Analysis of why a pill beats an injection into the brain. Read the details.
RNA Editing: Breakthrough in Nature and a New Class of Drugs
Breakthrough in RNA Editing: Nature Data on Cas13b-ADAR2 and the MIRROR System. The RNA Therapy Market Is Growing. Read About the New Class of Drugs and Treatment Prospects.
Genetically modified virus stopped pancreatic cancer: breakthrough
Oncolytic adenovirus from the University of Minnesota halted tumor growth in three patients with pancreatic cancer. Learn about the mechanism, risks, and prognosis.
Optic nerve regeneration: gene therapy and stimulation
Nature breakthrough: optic nerve regeneration in mice using gene therapy and visual stimulation. Analysis of implications for glaucoma treatment and the market. Learn more.
Cholesterol gene therapy VERVE-102: NEJM data, LDL reduction by 62%
NEJM published phase 1b results of gene therapy VERVE-102: LDL reduction by 62% after a single injection. Analysis of efficacy, risks, and prospects for patients with hypercholesterolemia. Read more.
Targeted therapy for Usher syndrome 1B: new data on AAVB-081
Interim data from phase 1/2 gene therapy AAVB-081 for Usher syndrome 1B: safety, efficacy, and prognosis. Learn about the dual AAV vector and treatment prospects.
Rett Syndrome: Access to Taysha Gene Therapy for a Girl from Ontario
The story of Lucia Vaccaro's family seeking access to gene therapy for Rett syndrome. Challenges of clinical trials, ethics, and regulatory barriers. Learn the details.
AAV9 gene therapy for SMA: long-term efficacy study
A long-term study in Nature Communications confirmed the efficacy of optimized AAV9 gene therapy for spinal muscular atrophy. Learn about safety and treatment prospects.
Gene therapy AB-1009 for Pompe disease: clinical trial
A clinical trial of gene therapy AB-1009 for late-onset Pompe disease (LOPD) has begun. Phase 1 analysis, mechanism of action, risks, and prognosis. Learn more.
ATF2 gene: key CRISPR target for neuron rescue
Neuron study: CRISPR knockout of ATF2 gene prevents neuron death during chemotherapy, ALS, and glaucoma. Analysis of a breakthrough in neuroprotection. Learn more.
Vyjuvek gene gel treatment butterfly disease DEB 2026
UK approves Vyjuvek gene gel for wound treatment in dystrophic epidermolysis bullosa. Efficacy 67% at 6 months. Learn mechanism of action and prospects.
AI-designed viral vectors: 50-fold increase in gene delivery to the brain
WhiteLab Genomics created AAV vectors with AI that deliver genes to the mouse brain 50 times more efficiently and accumulate less in the liver. Learn about the breakthrough in gene therapy.
Marne-Cel FDA approval: gene therapy for LAD-I
FDA approved Marne-Cel for children with severe LAD-I: 100% survival in trials. Learn how Rocket Pharmaceuticals turned rejections into a breakthrough.
Breakthrough Prize 2026 Laureates: Gene Therapy and CRISPR
Analysis of Breakthrough Prize 2026 Laureates: How FDA-Approved Gene Therapy and CRISPR Editing Became a New Business Model and What Awaits ALS Therapy. Learn About the Main Trends.
Gene therapy against aging: hunting the RAGE gene
Russian scientists are creating the first gene therapy that blocks the aging receptor RAGE. Learn how turning off the gene can prolong cell youth. Project details.
Personalized CRISPR therapy saved an infant: in vivo breakthrough
A child with the ultra-rare CPS1 mutation survived thanks to personalized CRISPR therapy in 6 months. Learn about the breakthrough in genetic engineering and N-of-1 platforms.
Gene therapy for OTOF deafness: hearing restoration
FDA approved the first gene therapy for hereditary deafness Otarmeni. Learn how a single injection restores hearing in OTOF mutation and changes the market.
ASCO 2026: biomarkers of response to Reqorsa gene therapy for lung cancer
Reqorsa gene therapy identified TROP2 and PTEN biomarkers for lung cancer prognosis. Learn ASCO 2026 data and prospects for treating NSCLC and SCLC.
Gene therapy OPGx-LCA5 accepted into FDA RDEP program
Learn how Opus Genetics' gene therapy OPGx-LCA5 received RDEP status from the FDA, accelerating the development of treatment for ultra-rare LCA5 blindness in children. Detailed analysis and prospects.
FDA approved first gene therapy for LAD-I in children
FDA approved Kresladi (marnetegragene autotemcel) — the first stem cell gene therapy for children with severe LAD-I. Learn about the mechanism, precedents, and business model. Detailed analysis on the website.
Gene therapy against aging: Russian RNA drugs block RAGE
Scientists from Russia are creating the world's first gene therapeutic drug for aging: a RAGE gene blocker and exosomes against sarcopenia. Learn how the state project promises to restore muscles and prolong active life.
EMA approved SMA gene therapy Itvisma and a drug for MS
European regulator recommended Itvisma for adults with SMA and Cenrifki for secondary progressive sclerosis. Learn how a one-time injection and a pill fill gaps in neurodegeneration treatment.
FDA approved the first gene therapy for hereditary deafness
Regeneron's Otarmeni restores hearing to normal in 42% of patients. The therapy treats OTOF gene mutations with a single injection. Learn how AAV vector technology has for the first time restored the ability to hear a whisper.