UniQure reported its gene therapy for Huntington's disease showed diminished benefits at four years compared to three years in a small trial, with shares plunging 48% as the results cast doubt on the treatment's long-term efficacy.
Beacon Therapeutics reported its experimental gene therapy improved low-light reading ability in a late-stage trial for X-linked retinitis pigmentosa (XLRP), a rare inherited eye disease that can cause blindness. The company succeeded where larger drugmakers including Johnson & Johnson and Biogen have failed.
The FDA granted full approval to Ultragenyx's gene therapy Fayuvi for Sanfilippo syndrome type A, marking the first-ever approved treatment for the ultra-rare, fatal neurodegenerative disease. The approval comes after a previous regulatory rejection and represents a significant milestone for patients with this devastating condition.
Dutch biotech UniQure submitted its Huntington's disease gene therapy to the FDA for approval after the agency reversed its earlier position on the experimental treatment, following an unusually turbulent development process.
The FDA halted testing of Regenxbio's gene therapy for Hunter Syndrome after spinal MRI scans detected abnormalities in five trial participants. This is the second clinical hold placed on a Regenxbio gene therapy program.
PTC Therapeutics won the bankruptcy auction for Sangamo Therapeutics' gene therapy assets with a $211 million bid. Sangamo filed for Chapter 11 bankruptcy in June after failing to find strategic alternatives.
Skylark Bio has dosed the first patient in its SONIX Phase 1/2 trial of SKY-GJB2, a gene therapy targeting GJB2-related hearing loss, the most common genetic cause of pediatric deafness. The trial follows earlier breakthrough gene therapy treatments that restored hearing in children born deaf.
Intellia Therapeutics announced second quarter 2026 financial results alongside positive Phase 3 clinical data for lonvo-z in hereditary angioedema (HAE), with FDA biologics license application acceptance anticipated in late 2026 and U.S. commercial launch planned for first half of 2027.
Catherine L'Estrange from North Acton became the first patient in the UK and second in the world to receive groundbreaking gene therapy for Bardet-Biedl syndrome, a rare condition that progressively causes blindness in children. The treatment, performed at St Helier Hospital in March, involves injecting healthy copies of a gene directly into the eye.
Three major research institutions announced the creation of the Center for Therapeutic Genetics, a nonprofit collaboration aimed at developing standardized genetic medicines, including base and prime editing, for rare and ultra-rare diseases that currently lack treatments.
Two patients with SCN2A-related developmental epileptic encephalopathy experienced reductions in seizure frequency after receiving individualized allele-selective antisense oligonucleotides. The treatments were well tolerated, with no ASO-related serious adverse events reported in two parallel single-patient studies published in Nature Medicine.
The FDA has approved Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel) for patients aged two years and older with sickle cell disease and transfusion-dependent beta thalassemia, expanding from the previous minimum age of 12 years. The approval was granted under the agency's Commissioner's National Priority Voucher program.
The FDA indicated it would reverse its earlier rejection of Regenxbio's gene therapy for a deadly childhood disease, stating that existing data would be sufficient to support an accelerated approval bid. The reversal represents a significant policy shift.
Doctors have directly linked a pediatric patient's tumor to gene therapy treatment viruses, marking a rare documented case of this safety risk. The finding comes as the gene therapy field grapples with safety-efficacy tradeoffs, with separate research showing that drugs designed to improve gene therapy safety may reduce effectiveness.
Five years after a disastrous trial where some boys died while others had astonishing recoveries, Astellas is returning with a revised gene therapy treatment for X-linked myotubular myopathy (XLMTM), offering new hope to a devastated rare disease community.
Researchers at Duke University are developing a gene therapy treatment designed to help repair heart damage following heart attacks, potentially changing how doctors treat cardiac injury from the leading cause of death in the U.S.
Intellia Therapeutics announced that its CRISPR-based gene editing treatment reduced swelling attacks in a pivotal trial for hereditary angioedema, a rare genetic disease. The results advance the CRISPR field's one-and-done treatment approach toward potential regulatory approval.
The FDA has greenlit a first-of-its-kind gene therapy to treat a rare form of hereditary hearing loss, marking a breakthrough that could pave the way for other hearing impairment treatments. Follow-up research confirms the therapy yields lasting hearing gains for patients with inherited deafness.
International clinical trial results show that gene therapy for a rare form of genetic deafness successfully restored hearing in 90% of participants, with improvements lasting years after treatment. The study was co-led by Mass General Brigham and China's Eye & ENT Hospital of Fudan University.
The 2026 Breakthrough Prize honored six laureates with $3 million awards for discoveries including gene therapies for inherited blindness and sickle cell disease, as well as groundbreaking work on KRAS cancer targets. Boston-area researchers were among those recognized.
