Gene Therapy

Two investigational therapies for Rett syndrome, using different technologies, aim to control expression of the delivered gene and avoid the adverse events associated with conventional gene therapy.

Paying in installments with adjustments according to how well a gene therapy works is one way payers are coping with therapies with list prices in millions of dollars.

A new gene therapy administered directly to the brain aims to preserve dopamine neurotransmission in patients with multiple system atrophy.

Payers must be early adopters of managing genetic testing through DEX Z-Codes, combined with science-based policies and policy adherence and claims editing technology.

Sarepta Therapeutics plans to submit an application for full approval of Elevidys to treat all ages of patients with Duchenne muscular dystrophy. Company officials said the FDA is open to evaluating the application based on the totality of the evidence.

Many carriers reported feelings of concern, anxiousness, and guilt for passing the X-linked inherited retinal disease to their children—and 78% of respondents in a new study believe that carriers should have access to gene therapy options.

RP-L201 (marnetegragene autotemcel) is a one-time gene therapy that delivers a functional copy of the ITGB2 gene, which provides instructions for immune response to infections. The Prescription Drug User Fee Act (PDUFA) target action date is March 31, 2024.

Researchers at the Yale School of Medicine have invented a new delivery technology that has the potential to create one-time treatments for a range of neurological disorders.