Regeneron will make its newly approved gene therapy available for free in the U.S. The company hasn't yet decided how much it ...

Jessica Rendall is a reporter with experience covering a variety of health and wellness topics, including chronic disease, infectious disease, telemedicine, mental health and health technology. Kelly ...

In utero gene therapy uses prenatal genetic screening to identify fetuses with lethal mutations and delivers corrective genes ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious and common complication of the condition, has shown promising results in a ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

This voice experience is generated by AI. Learn more. This voice experience is generated by AI. Learn more. Miles, age 2, is one of the first patients at Boston Children's hospital to receive gene ...

Individualized gene therapies reduce seizures and lead to developmental gains in two boys with severe, treatment-resistant SCN2A-related epilepsy.

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Fresh with a rebrand, Cyllene will advance gene therapies built on a platform that uses a modified form of the herpes virus for delivery.