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If approved, Regeneron's Otarmeni will be the first gene therapy for OTOF-related hearing loss in the European Union.
In May 2025, a young boy received an injection delivering a healthy copy of a gene called OTOF into the cochlea of his inner ear — one of the first patients with hereditary hearing...
Sensorion is focusing on SENS-601 targeting GJB2 mutations, while discontinuing development of its OTOF-targeting program, SENS-501.
Med-El has acquired two gene therapy programs from Rescue Hearing Inc targeting mutations in the MYO7A and STRC genes, expanding the hearing implant company's pipeline into biologi...
A 6-year-old boy with Cockayne syndrome — a rare genetic disorder that causes hearing loss, vision impairment, and early death — has become the first child to receive an experiment...
A strategic review determined that the development environment had "notably changed," leading Sensorion to end work on its gene therapy for OTOF-related hearing loss.
Critical Path Institute Awards $249,719 Grant to Odylia Therapeutics for Groundbreaking Gene Therapy Targeting Usher Syndrome Type 1C Vision Loss In an ambitious effort to combat a...
An 8-month-old infant with severe genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of...
Travis Smith was 1 year old when he underwent surgery on both ears. He's now babbling, dancing and laughing.
The funding deal is a vote of confidence in the sales potential of multiple prospects in late-stage development — one of which MeiraGTx recently reacquired from J&J.
By combining closed-loop functional screening with AI-driven design, Bonito and DaltonTx aim to accelerate discovery of delivery systems that can reach tissues and cell types that...
In the swiftly evolving landscape of gene therapy, antibody-oligonucleotide conjugates (AOCs) have emerged as a transformative platform primed to revolutionize targeted molecular d...
The ProHear Study found that the drug candidate Bimokalner produced a clinically meaningful reduction in pure-tone audiometry threshold shifts in testicular cancer patients who dev...
You might recall that gene therapy to overexpress caveolin-1 in the brain was recently shown to reduce pathology in a mouse model of Alzheimer's disease. In today's open access pap...
As MeiraGTx lines up the pieces for a commercial push, the company has new funding to work with.
The company's proprietary platform combines an ultrasound-mediated delivery technology with payload engineering capabilities that support the development of DNA and RNA therapeutic...
In a groundbreaking study that could redefine treatment paradigms for spinal muscular atrophy (SMA), researchers have released a comprehensive long-term comparative analysis of ade...
An experimental gene therapy called SynCav1 can protect the central nervous system from the cognitive decline linked to TDP-43-related proteinopathy, a primary driver of FTD, ALS,...
A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hosp...
The data showed that del-brax, an “antisense oligonucleotide conjugate” being tested against a muscle-wasting disease, met its primary endpoint in a Phase 1/2 study.
A new treatment platform developed by researchers at the University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into pre...
Sanofi and Wave Life Sciences are using their time at the annual meeting of the American Thoracic Society in Orlando to share updated data for their next-gen attempts to treat a pr...
A gene therapy aimed at correcting fragile X syndrome by restoring the missing FMR1 protein has demonstrated multiple disease-relevant improvements in a mouse model, according to a...
The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of el...
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