gene therapy

The FDA approved gene therapy for treating sickle cell disease in children aged 2 and older. A Nashville doctor described the approval as a medical milestone.

The FDA has approved a new gene therapy treatment for children aged 2 or older with sickle cell disease. This marks a significant advancement in managing the condition.

An individual received a ₹35 LPA job offer for an Executive Assistant/Founder support role at Shya Exports, involving gene therapy and glutamine therapy, with an international employment arrangement. The candidate is seeking advice due to the unusual nature of the opportunity.

Dr. Thein Swee Lay, the only Malaysian scientist to win the Breakthrough Prize, discusses her work in gene therapy and nostalgia for her hometown Kuantan's popiah in an exclusive interview. She highlights challenges finding authentic popiah in the US while reflecting on her Malaysian roots.

A 23-year-old New Orleans man became the first in Louisiana to be functionally cured of sickle cell disease through gene therapy. Daniel Cressy underwent the treatment at Manning Family Children’s Hospital and now plans to establish a nonprofit to support others with the disease.

A 23-year-old New Orleans man, Daniel Cressy, became the first person in Louisiana to be functionally cured of sickle cell disease through gene therapy. The procedure, conducted at Manning Family Children’s Hospital, involved editing his stem cells to eliminate the defective cells causing the disease, with Cressy now planning to support others through his nonprofit.

Daniel Cressy, a 23-year-old from Louisiana, is the first in the state to be functionally cured of sickle cell disease via gene therapy at Manning Family Children’s Hospital. The procedure used his own edited stem cells, and he aims to launch a nonprofit to support others undergoing similar treatment.

The FDA has agreed to reconsider a gene therapy for Hunter syndrome developed by Regenxbio after initially rejecting it due to concerns about clinical trial design. The agency is now open to reviewing existing data rather than requiring new trials, reflecting a broader trend in regulatory flexibility for rare-disease treatments.

Homing pigeons may navigate using magnetic immune cells in their liver. The article also mentions a gene therapy trial to restore heart muscle and a reptile museum in Ecuador.

Eli Lilly's gene therapy reduced cholesterol by 62% in a Phase 1 trial, with no serious adverse events. The company acquired the therapy through its $1 billion buyout of Verve Therapeutics and plans Phase 2 trials. Lilly also agreed to buy three vaccine developers (Curevo, LimmaTech Biologics, and Vaccine Company) in deals totaling up to $4 billion, expanding its focus on infectious diseases.

Immunovant shares surge following positive arthritis trial data. UCSF researchers have submitted an FDA application for a prenatal gene therapy targeting a severe neurodegenerative disorder affecting young children. FDA leadership instability is causing investor concern and impacting rare disease drug development.

A rare case of brain cancer in a child may have been caused by a virus administered during gene therapy treatment for a genetic disorder. Genetic analysis uncovered the connection between the therapeutic virus and the tumor development. The case raises important questions about the safety profile of gene therapy treatments.

A wearable robot is helping children with spinal muscular atrophy regain muscle strength, particularly those undergoing gene therapy. The device aids in muscle recovery for this rare neuromuscular condition. This innovation offers new hope for patients with this debilitating disease.

Scientists have discovered a set of genes that could help humans regrow lost limbs by studying axolotls, zebrafish, and mice. The researchers identified powerful SP genes involved in regeneration and used gene therapy to partially restore regeneration in mice. This breakthrough marks a major step toward future treatments that could replace damaged limbs with living tissue instead of prosthetics.

The FDA has approved the first-ever gene therapy for treating genetic hearing loss, marking a significant advancement in medical science. The therapy was authorized under the FDA's National Priority Voucher program, which expedites treatments for rare diseases.

President Donald Trump highlighted 2-year-old Travis Smith, who regained hearing after a gene therapy treatment by Regeneron. The FDA granted accelerated approval for Regeneron's Otarmeni, a gene therapy administered surgically, which the company will provide free in the U.S. Trump also noted Regeneron's agreement to offer discounted drug prices.

A breakthrough gene therapy drug, Otarmeni, has received FDA approval and will be provided free to American children with total hearing loss. This development marks a significant advancement in treating congenital hearing impairment through gene therapy.

The FDA approved a gene-therapy drug developed by Regeneron to treat genetic hearing loss, marking the first such therapy for this condition. This approval represents a significant advancement in medical treatments for hereditary hearing impairments.

The FDA has approved the first gene therapy for deafness, developed by Regeneron Pharmaceuticals, marking a significant medical milestone for treating a rare form of the condition.

Gene therapy for a rare type of deafness enabled children and adults up to 32 years old to hear for the first time, with some patients experiencing lasting benefits for over two years.

The 2026 Breakthrough Prizes awarded US$3 million to researchers in magnetic muon measurements and gene-therapy advancements. Hundreds of physicists from over 30 institutions were recognized for their contributions.

A new gene therapy has restored hearing in ten patients born deaf within weeks of a single injection directly into their inner ear.