Caeregen Therapeutics, a regenerative medicine therapeutics company and Wacker Biotech, a contract development and manufacturing organization (CDMO), will collaborate on the development and production of CTR-107 (Noregen), a novel regenerative therapeutic for the treatment of retinal-related vision loss. As Caeregen’s CDMO partner for CTR-107, Wacker Biotech will produce drug substance at its site in Halle, Germany, and complete clinical trial drug product production at its site in Amsterdam, the Netherlands, to support Phase I/II clinical study initiation in 2024.

CTR-107 is the first program to obtain U.S. FDA Rare Pediatric Disease Designation for treatment of Familial Exudative Vitreoretinopathy (FEVR).

CTR-107 is a synthetic targeted growth-factor that mimics the properties of norrin, a naturally occurring human protein that promotes development of normal, organized blood vessels and neurons in the human eye, ear, and central nervous system. CTR-107 is being developed for the treatment of Familial Exudative Vitreoretinopathy (FEVR), a genetic disorder of retinal blood vessel formation, resulting in partial or complete vision loss.

Noregen received Rare Pediatric Disease designation from the U.S. FDA for the treatment of Familial Exudative Vitreoretinopathy (FEVR), rendering Caeregen eligible to obtain a Priority Review Voucher at the time of marketing approval. The RPD designation for Noregen is the first to be granted by FDA for children with FEVR and follows the attainment of Orphan Drug Designation (ODD) by both the FDA and European Commission European Medicines Agency (EMA).

Caeregen first contracted Wacker Biotech in 2022 to support initial manufacturing and process development for CTR-107 and has now partnered with Wacker Biotech to manufacture active CTR-107 drug substance at its site in Halle, Germany, for the first planned Phase I/II clinical study. Finished CTR-107 clinical study drug product manufacturing will be completed at Wacker Biotech’s site in Amsterdam, the Netherlands. Production in compliance with pharmaceutical GMP (Good Manufacturing Practice) requirements is scheduled to start in early 2024.

“As a potential breakthrough program for a rare, devastating retinal disease with no therapeutic options for patients, we selected Wacker Biotech as our CDMO partner for CTR-107 development because of their long-standing experience and deep understanding of protein chemistry, and shared urgency in advancing transformative science,” said Walter Capone, CEO of Caeregen Therapeutics.

“Through its unique structure, CTR-107 has the potential to repair and regenerate the neurovasculature to restore vision, with potential applications as well to reverse hearing loss and treat neurodegenerative diseases,” said Guido Seidel, vice president business unit BioPharma at WACKER. “We are pleased to support Caeregen Therapeutics in the development of this revolutionary regenerative drug candidate and product platform,”