Andelyn Biosciences and Odylia Therapeutics Partner to Manufacture Novel Gene Therapy for Vision Loss

News & MediaFebruary 1, 2023

Novel AAV Capsid Anc80 enables efficient gene transfer

COLUMBUS, OH, 31st JANUARY, 2023Andelyn Biosciences, Inc., a pioneering and patient-focused gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Odylia Therapeutics, a nonprofit 501(c)(3) biotech focused on accelerating rare disease drug development, to manufacture a novel gene therapy (OT-004) to treat vision loss caused by mutations in the RPGRIP1 gene.

Mutations in the RPGRIP1 gene cause rapid decline in function of the photoreceptor cells of the retina, resulting in a rare type of vision loss that can begin as early as infancy. Patients are usually diagnosed with one of three forms of retinal dystrophy: Leber Congenital Amaurosis 6, Cone Rod dystrophy 13, or juvenile Retinitis Pigmentosa. OT-004, is an investigational AAV gene therapy that utilizes the Anc80 AAV vector for efficient gene transfer of RPGRIP1 to treat vision loss in patients lacking a functional copy of the gene.

Andelyn and Odylia’s partnership grew out of a long-standing relationship. Following a review of the gene therapy manufacturing marketplace and impressed by Andelyn’s work within the rare disease space, Odylia recognized that Andelyn had the unique capabilities to manufacture this novel AAV capsid Anc80 and bring the treatment to clinical trials.

Wade Macedone, Chief Operations Officer at Andelyn, said: “At Andelyn, we are committed to making an impact on the lives of patients affected by rare diseases. We have the expertise and are determined to support Odylia in bringing this new therapy to clinical trials to give hope to patients with RPGRIP1-associated vision loss.”

Ashley Winslow, President and Chief Scientific Officer at Odylia Therapeutics, said: “There is currently no treatment for vision loss caused by RPGRIP1 mutations. Finding the right partner to manufacture our gene therapy was critical to the success of the program. Andelyn is well respected in the rare disease space and recognizes where we are, where we came from, and where we are headed.”

Andelyn recently expanded and now operates in three facilities for plasmids, preclinical, and GMP clinical and commercial manufacturing.  The opening of its new Andelyn Corporate Center completes a network of flexible and scalable sites that support end-to-end gene therapy contract development and manufacturing.


Contact information
Vera Araujo, Sr. Director, Sales and Strategic Partnerships

For Andelyn media inquiries, please contact:
Imogen Quail, PR Manager

For Odylia Therapeutics media inquiries, please contact:
Ellynn Szoke, Managing Director


About Andelyn Biosciences, Inc.
Andelyn is a biopharmaceutical CDMO with a mission of Pioneering Solutions That Turn Hope into Reality™. Headquartered in the greater Columbus, Ohio, USA area, Andelyn’s capabilities span viral vector process and analytical development, small to large-scale adherent and suspension-based GMP drug substance manufacturing up to 2000L, and drug product manufacturing services. Having 20+ years of experience in viral vector manufacturing, Andelyn offers its clients direct access to globally recognized thought leaders that have enabled 75+ worldwide clinical trials. With the experience gained from over 400 cGMP clinical batches and 2000+ research-grade productions, Andelyn provides its clients with end-to-end manufacturing services, advanced quality systems, full regulatory support, and supply chain vertical integration. Andelyn began operations in its state-of-the-art 185,000 sq. ft. commercial manufacturing facility in September 2022, expanding capacity across 14+ production suites for customization of new programs and tech transferred programs. Andelyn is one of a few select CDMOs that offer clinical through commercial-scale capabilities that will help fulfill Andelyn’s mission of accelerating the development and manufacturing of innovative therapies to bring more treatments to more patients. Andelyn is owned and supported by Nationwide Children’s Hospital and Pall-Cytiva, part of Danaher Corporation.

About Odylia Therapeutics
Odylia Therapeutics is a nonprofit 501(c)(3) organization focused on accelerating drug development for rare and ultra-rare diseases. Odylia has disclosed two gene therapies currently in preclinical development. The lead program, OT-004, is an investigational AAV gene therapy that utilizes the Anc80 AAV vector for efficient gene transfer of RPGRIP1 to treat vision loss in patients lacking a functional copy of the RPGRIP1 gene. Leveraging internal expertise, Odylia also works with patient groups, industry, and academic researchers to advance their rare disease efforts.

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