Agilis Biotherapeutics and Waisman Biomanufacturing Enter Into Exclusive Manufacturing Agreement for Friedreich’s Ataxia Gene Therapy

Waisman to provide global supply of Agilis' novel AGIL-FA gene therapy

CAMBRIDGE, Mass. & MADISON, Wis.--(BUSINESS WIRE)-- Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative gene therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman Biomanufacturing, a non-profit gene and cell therapy development and manufacturing group located at the UW-Madison Waisman Center, (Waisman) announced today that the companies have entered into an exclusive partnership agreement for the production of Agilis' novel gene therapy product, AGIL-FA, for the treatment of Friedreich's ataxia (FA). FA is a debilitating multi-system disease arising from mutation of the FXN gene. It is the most common inherited ataxia, with 1 in every 100 people being carriers of a mutated FXN gene. FA typically arises between the ages of 5 and 15 and manifests as difficulty with balance and coordination. Over time, the disease progresses to an array of neurological symptoms and life-altering changes in mobility, energy, speech, hearing, and other body systems including the cardiovascular system, which collectively reduce longevity in many cases.

Following the completed evaluation and selection of the lead therapeutic candidate for FA, AGIL-FA, Agilis has completed molecular characterization and initial proof-of-concept and biodistribution studies of AGLIL-FA in advance of submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration. The FXN gene construct used in AGIL-FA was optimized and developed in partnership with Intrexon Corporation (NYSE: XON).

Under the terms of the agreement, Waisman will utilize its personnel, facilities and proprietary processes to manufacture GLP material for non-clinical studies, cGMP material for non-clinical and clinical studies, and potential future commercial supply should AGIL-FA be successfully developed and approved. Agilis and Waisman will each provide team experts to contribute to the overall execution of the full range of manufacturing, quality and regulatory activities.

"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," said Dr. Mark Pykett, Agilis President and CEO. "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."

Founded in 2001, Waisman Biomanufacturing, a non-profit entity of the Waisman Center and the University of Wisconsin, currently operates a 15,000 square foot biologics manufacturing facility with eight cGMP compliant cleanroom areas to accommodate clinical production of mammalian and microbial therapies and aseptic filling of final products. The Waisman Quality System and cleanroom facility are designed to maximize regulatory compliance and environmental quality.

Dr. Derek Hei, Director of Waisman, commented, "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."

About Friedreich's Ataxia

Friedreich's ataxia (FA) is an inherited neuromuscular disorder most commonly caused by a single genetic defect in the FXN gene that leads to reduced production of frataxin, a mitochondrial protein that is important for iron metabolism. FA results in a physically debilitating, life-shortening condition and is the most common hereditary ataxia, with an estimated 5,000 to 10,000 patients in the U.S. (i.e., one in every 50,000 people). Both male and female children can inherit the disorder. Symptoms of FA include progressive loss of coordination and muscle strength, which lead to the full-time use of a wheelchair; scoliosis (which often requires surgical intervention); diabetes mellitus; hearing and vision impairment; serious heart conditions; and premature death. Current FA therapies are primarily focused on symptomatic relief, and there are no FDA-approved drugs to treat the cause of FA.

About Agilis Biotherapeutics

Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis' therapies are engineered to impart sustainable clinical benefits, and potentially a functional cure, by inducing persistent expression of a therapeutic gene. The Company's technology is aimed at the precise targeting and restoration of a lost gene function, while avoiding unintended off-target effects. Agilis' integrated strategy increases the efficiency of developing DNA therapeutics into safe, targeted gene therapies that achieve long-term efficacy and enable patients to remain asymptomatic without continuous invasive treatment. Agilis' rare disease programs are focused on gene therapy for AADC Deficiency, Friedreich's ataxia, Angelman syndrome, and Fragile X syndrome, rare genetic diseases that include severe neurological deficits and result in physically debilitating conditions.

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About Waisman Biomanufacturing

Waisman Biomanufacturing ( is a non-profit biotherapeutics development and manufacturing group that is part of the UW-Madison Waisman Center. The mission of WB is to accelerate the advancement of promising biotherapeutics into human clinical trials through collaborations with academic investigators and biotechnology companies. WB has extensive experience in cGMP production of biologics for phase I/II human clinical trials including: viral vectors and vaccines, plasmid DNA, recombinant proteins, and cell therapeutics. WB provides comprehensive services to address key issues for early-stage projects including: technology transfer, process development, assay development, process qualification/validation, assay qualification/validation, cGMP manufacturing of pre-clinical and clinical material, quality control testing and aseptic filling. In addition, WB offers full support for regulatory filings.

Safe Harbor Statement

Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

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Agilis Biotherapeutics
Dr. Jodi Cook, 510-673-7809
Vice President, Operations & Strategic Alliances

Source: Agilis Biotherapeutics, LLC

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