Agilis Biotherapeutics, LLC, and Waisman Biomanufacturing have signed an exclusive partnership agreement to develop and produce AGIL-FA, Agilis’ potential gene therapy for the treatment of Friedreich’s ataxia (FA).
Agilis, a biotechnology company specializing in therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman, a nonprofit gene and cell therapy development and manufacturing group, worked to develop and optimize the FXN gene construct used in AGIL-FA in partnership with Intrexon Corporation. AGIL-FA has now completed molecular characterization, and initial proof-of-concept and biodistribution studies, and Agilis is preparing to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA).
“Our partnership with Waisman is an important step in advancing our AGIL-FA gene therapy for the potential treatment of the neurological symptoms in Friedreich’s ataxia patients,” Dr. Mark Pykett, Agilis’ president and CEO, said in a press release. “Waisman is a leading manufacturer of biological products, with extensive experience and a strong track record in the production of innovative medical products. In partnering with Waisman to leverage their proprietary systems, organizational expertise, and extensive infrastructure, Agilis has solidified a key strategic component in the near-term and long-term development and commercialization of AGIL-FA. We are pleased to partner with such a reputable organization as Waisman to ensure high quality, scalable manufacturing of the product.”
Under the agreement’s terms, Waisman will provide staff, services and proprietary processes to manufacture all needed GLP (good laboratory practice) material and cGMP (current good manufacturing practice) material for nonclinical and clinical studies, and potential future commercial supply of AGIL-FA if the product is effectively developed and approved. Both companies will place their experts at this project’s disposal to accelerate manufacturing and quality and regulatory actions.
“Our partnership with Agilis is reflective of Waisman’s mission to provide high quality cGMP biologic products to our partners and to assist with the advancement of innovative medicines to enhance the treatment of human diseases. We are pleased to collaborate with Agilis on its exciting gene therapy product for Friedreich’s ataxia and to facilitate supply of the product for the sequence of development stages required for its approval and ultimate commercialization,” said Dr. Derek Hei, director of Waisman, a nonprofit entity of the Waisman Center and the University of Wisconsin.