Agilis Biotherapeutics has been awarded a Cooperative Research and Development Agreement by National Center for Accelerating Translational Sciences (NCATS) to facilitate development of the company’s gene-therapy candidate for aromatic L-amino acid decarboxylase (AADC) deficiency.

The value of the award was not disclosed, though Agilis said the partnership “could potentially be worth up to several million dollars to Agilis in in-kind services and activities from NIH.”

Agilis said it joined NCATS in signing a letter of intent for the CRADA, which was awarded under NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program.

TRND is designed to use NIH resources to speed up development of new treatments for diseases with unmet medical need. Agilis is the first gene therapy company to receive a CRADA through TRND.

Agilis’ collaboration with NCATS includes conducting toxicology, process development, and manufacturing work necessary for overall development of the gene therapy, as well as registration in the U.S. and abroad.

Agilis has licensed its AADC deficiency gene therapy program from National Taiwan University (NTU) based on research by Paul Wuh-Liang Hwu, M.D., Ph.D., at NTU Hospital, where 18 patients have been treated.

Following a single administration of the gene therapy, according to Agilis, treated patients showed substantial, durable gains in motor and cognitive function over multiple years, exhibited de novo production of dopamine as visualized by F-DOPA positron emission tomography (PET) imaging, and realized improvements in metabolic biomarkers.

The University of Florida Powell Gene Therapy Center was instrumental in developing the initial product manufacturing and toxicology work in preparing the program for clinical trials, Agilis added.

In addition to AADC deficiency, Agilis’ rare disease programs are focused on developing gene therapies for Friedreich’s ataxia, Angelman syndrome, and Fragile X syndrome.

“Our longer-term goal is to engineer, develop, and commercialize a robust pipeline of pioneering DNA therapeutics that address a broad range of rare CNS diseases for which there are no or limited therapies, and for which the basis for biomedical intervention is clear,” Agilis states on its website.

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