Our Partners
With your help, we have assembled a dream team of scientists, clinicians, advisors and drug development experts to guide our efforts. This group shares our enthusiasm and our optimism about bringing a successful therapy to life. Together, we will stop this blinding eye disease in its tracks!
Our Bio-Tech Partners
The NPHP1 Family Foundation is proud to be partnering with non-profit biotech, Odylia Therapeutics to develop a gene replacement therapy for vision loss caused by mutations to or deletions of the NPHP1 gene.
Learn more about Odylia: www.odylia.org
Our Pre-Clinical Partners
Together with Odylia, the NPHP1 Family Foundation has partnered with the lab of Dr. Friedhelm Hildrebrandt at Harvard University’s Boston Children’s Hospital to test an AAV-based therapy for NPHP1 retinal dystrophy on two different mouse models.
This work follows a successful gene replacement therapy proof of concept conducted in the lab of Dr. Lingzhong Sun, at Southern Medical University in Guangzhou China. Dr. Hildebrandt’s team is currently conducting the natural history, pharmacology and efficacy studies, with a goal of having initial data prepared by the end of 2025. Following these preclinical studies and manufacturing activity, Odylia is preparing for IND submission in 2027.
The NPHP1 Family Foundation, through our partnership with Odylia, is working with Powered Research, a North Carolina based contract research organization that specializes in preclinical research in ophthalmology and oncology. Powered Research is performing critical mouse studies to determine the dosing and efficacy of our therapy.
VectorBuilder is the world’s largest provider of custom vectors for both viral and non-viral gene delivery and is a trusted partner for thousands of labs and biotech/pharma companies around the world. The NPHP1 Family Foundation is thrilled to be among that number and has partnered with VectorBuilder to advance the research-grade version of our therapy.
Our Clinical Partners
Through our co-development partnership with Odylia Therapeutics, the NPHP1 Family Foundation is thrilled to have Philadelphia, PA-based Puresyn on our team. For more than 30 years, Puresyn has partnered with gene and cell therapy innovators to provide high-quality plasmid DNA backed by scientific precision and built on trusted relationships.
We have research collaborators and interest from biotech partners ready to develop an NPHP1 gene therapy. We’ve also identified a small population of people from around the world who will be impacted by this treatment. We have so many tailwinds behind us.
We need you to join the cause.
Your donation is 100% tax-deductible.