Puresyn, Odylia Therapeutics, and the NPHP1 Family Foundation Announce Manufacturing Partnership to Advance an N-of-1 Gene Therapy Study for NPHP1-Associated Vision Loss

Public Announcement
FOR IMMEDIATE RELEASE

MALVERN, PA 20 October, 2026 – Puresyn, Odylia Therapeutics, and the NPHP1 Family Foundation are pleased to announce a strategic collaboration to support the development of a gene therapy for NPHP1-associated retinal dystrophy.

Through this collaboration, Puresyn will provide plasmid DNA manufacturing support for a first-in-human study within Odylia’s NPHP1 program. High-quality plasmid DNA is an essential starting material for producing the adeno-associated viral (AAV) vectors used to deliver therapeutic genes.

“This collaboration supports a first-in-human study focused on initial treatment of a single patient of an ultra rare disorder, making the purpose behind our work deeply personal,” said Michael Moxley, Director of Business and Process Development at Puresyn. “We’re excited to be part of this journey with Odylia and the NPHP1 Family Foundation, and hopeful that our contribution can help bring joy to this patient and his family, and future treated individuals living with NPHP1-associated retinal dystrophy”

We are deeply inspired by the families, scientists, and nonprofit organizations working to move rare disease therapies forward. Their commitment reinforces our own dedication to responsive collaboration, technical excellence, and reliable manufacturing support.

We’re proud to contribute our expertise to this shared effort and to work with Odylia and the NPHP1 Family Foundation toward a future with more treatment options for patients facing rare genetic diseases.

About Puresyn

Puresyn is a contract development and manufacturing organization specializing in plasmid DNA services, offering research-grade, INDReady®, and cGMP plasmid production supported by established quality systems and scalable manufacturing infrastructure. Puresyn supports biopharmaceutical and cell and gene therapy companies from early discovery through commercial development with a focus on reliability, technical excellence, and responsive collaboration.

About Odylia Therapeutics

Odylia is a nonprofit biotechnology organization dedicated to advancing treatments for rare diseases that are often underserved by traditional drug development models. Together with the NPHP1 Family Foundation, Odylia is developing a gene replacement therapy aimed at preventing vision loss caused by mutations in or deletions of the NPHP1 gene. Learn more at https://odylia.org.

About The NPHP1 Foundation

The NPHP1 Family Foundation brings the urgency and determination of the patient community to this effort, funding research to advance therapies that could preserve vision for affected children and adults. Learn more at https://www.nphp1.org.

Media Contact:
Puresyn:
Michael Moxley
Director, Business and Process Development
mmoxley@puresyn.com

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