FDA Approves CRISPR-based Therapeutic for Trial

Cure Rare Disease, a Boston-based nonprofit organization developing genetic medicines, has received approval from the Food and Drug Administration (FDA) to administer its CRISPR-based therapeutic that halts the progression of Duchenne muscular dystrophy.

Duchenne muscular dystrophy is a rare genetic disease caused by a mutation in the gene responsible for dystrophin production. It can affect all muscle types and leads to loss of mobility and eventually to respiratory and cardiac failure. The disease impacts 1 in 3,500 males and is extremely rare in females.

The drug, CRD-TMH-001, treats muscle promoter and exon 1 mutations on the dystrophin gene. With the FDA approval, dosing of the drug will take place at the University of Massachusetts Medical School very soon.

The therapeutic has been developed for one-time administration. It will be administered intravenously, and the patient will be under observation in the hospital for several days to ensure that there are no uncontrolled adverse reactions. As per FDA guidelines, the patient's progress will be tracked for 15 years.