Business:
Gene Therapy
Drug notes:
PBFT02 Clin1/Clin2 frontotemporal dementia; PBKR03 Clin1/Clin2 Krabbe disease; PBML04 Clin1/Clin2 metachromatic leukodystrophy; PBAL05 RD ALS; 2 undisclosed programs RD Huntington's disease, epilepsy
About:
Passage Bio is developing genetic medicines to treat rare disorders of the central nervous system. Drug development for neurodegenerative disorders has been challenging due to the blood brain barrier. Passage Bio’s solution is to create genetic drugs that are delivered directly into the cerebrospinal fluid, with the potential for long-term correction following a single treatment. Each genetic drug contains the functional therapeutic gene and the regulatory elements needed for the gene to be expressed. Passage Bio’s lead program is for people with frontotemporal dementia caused by a mutation in the GRN gene. The therapy involves delivering a functional GRN gene to elevate levels of progranulin in the brain.