Andelyn Biosciences will produce Odylia Therapeutics’ gene therapy, OT-004, which aims to treat vision loss caused by the RPGRIP1 gene.
Contract development manufacturing organization (CDMO) Andelyn has partnered with nonprofit biotech firm Odylia to produce OT-004, which is an investigational adeno-associated virus (AAV) that uses the ancestral AAV, Anc80 to create an efficient gene transfer of RPGRIP1 to treat vision loss in individuals that lack a functional copy of the gene.
“Andelyn’s adherent production platforms utilize a transient transfection of HEK293 cells in Corning HYPERStack (HS) vessel,” a spokesperson for Andelyn told us.
“Our production strategy is designed to accommodate the pooling of multiple sublots of upstream production into one bulk drug substance through multiple pool points during purification if needed. All-column purification will be performed prior to filling.”
According to the CDMO, itwas selected b after a review of its gene therapy production activity and work within the rare disease space. Both parties aim to manufacture OT-004 and bring the gene therapy to clinical trials.
“Odylia aims to initiate clinical trials for the RPGRIP1 Gene Therapy program (OT-004) in 2025,” the spokesperson 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,” said Ashley Winslow, president, and chief scientific officer at Odylia Therapeutics.
“Andelyn is well respected in the rare disease space and recognizes where we are, where we came from, and where we are headed.”
Andelyn opened a clinical and commercial manufacturing facility in Columbus, Ohio to support cell and gene therapy (CGT) development in October 2022. The firm said the plant has flexible and scalable facilities to support end-to-end gene therapy contract development and manufacturing.
The CDMO was also selected by INADcure Foundation to bring the first-ever gene therapy for Infantile Neuroaxonal Dystrophy (INAD) to clinical trials in December 2022.