Odylia Therapeutics, a US-based nonprofit (501(c)3) biotechnology company focused on advancing treatments for rare diseases, announced on Monday its latest pipeline project: a co-development partnership with the NPHP1 Family Foundation to create an AAV-based gene replacement therapy for retinal dystrophy caused by mutations in the NPHP1 (nephrocystin-1) gene.

The company also announced that it is working with collaborators at Boston Children's Hospital, in the lab of Dr Friedhelm Hildebrandt, an expert on NPHP nephrocystin genes and their associated ciliopathies. Dr Hildebrandt's team will be conducting the natural history, dosing and efficacy studies on two different NPHP1 mouse models -- one with a more aggressive retinal phenotype and one with a milder phenotype. The former mouse was used in a successful gene replacement therapy proof of concept in the lab of Dr Liangzhong Sun at Nanfang Hospital in Guangzhou, China.

Odylia says that, because rare conditions are believed to be of little to no commercial interest, its non-profit model creates a pathway for research and development that would otherwise not exist.

The NPHP1 project's preclinical work is planned to occur throughout the remainder of this year, and the team is targeting an Investigational New Drug (IND) submission and subsequent clinical trial in late 2027.