Tern Therapeutics Selected by US FDA to Participate in the CDRP Program to Expedite CMC Development of TTX-381 to Treat Vision Loss in CLN2 Disease
Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program is designed to speed CMC development of promising therapies to enable earlier patient access
TTX-381 was previously awarded Regenerative Medicine Advance Therapy (RMAT) and Fast Track Designations by the US FDA
Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced that TTX-381, the company’s investigational therapy to treat the ocular manifestations of CLN2 disease, has been selected by the U.S. Food and Drug Administration (FDA) to participate in the Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) Program. The CDRP program was created by the FDA to facilitate CMC readiness for a limited number of promising therapies with accelerated clinical development timeframes based on the anticipated clinical benefits of earlier patient access. Through the CDRP program, Tern plans to continue to collaborate closely with the FDA to expedite commercial manufacturing readiness of TTX-381.
“Our top priority is getting safe and effective therapies for CLN2 disease to patients as quickly as possible. Partnering with the FDA through the CDRP program will help ensure that late-stage commercial manufacturing for TTX-381 keeps pace with our accelerated clinical development,” said Alex M. Bailey, PhD, CEO of Tern.
FDA selected TTX-381 for the CDRP program based on three main criteria: (1) anticipated clinical benefits of earlier patient access to TTX-381, (2) novelty of TTX-381 and complexity of manufacturing processes, and (3) ability to align CMC development and readiness within the same overall clinical development timespan. Key benefits of the CDRP program include increased communication with the FDA via two additional CMC-focused Type B Meetings as well as opportunity for follow-up CMC-focused discussions, and an expansion of the FDA CMC and Quality teams supporting TTX-381 who are tasked with implementing science- and risk-based regulatory approaches. About TTX-381 TTX-381 is a novel one-time AAV gene therapy designed to deliver a working copy of the TPP1 gene directly to the retina to provide a durable source of TPP1 to maintain the health of the retina and address vision loss in people with CLN2 disease. Vision loss in CLN2 disease rapidly progresses to blindness, and there is currently no available treatment for the ocular manifestations of CLN2 disease. The open-label clinical trial to evaluate the safety and efficacy of TTX-381 is active and recruiting patients in an expansion cohort. Learn more at euclinicaltrials.eu (EUCT number: 2025-521175-31-00) and clinicaltrials.gov (NCT05791864).
