Agilis Biotherapeutics is advancing innovative DNA therapeutics designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Our therapies are engineered to impart sustainable clinical benefit, and potentially a functional cure, by inducing persistent expression of a therapeutic gene. The company’s technology enables precise targeting and restoration of lost gene function, while avoiding unintended off-target effects. Our integrated strategy increases the efficiency of developing DNA therapeutics to more rapidly advance safe, targeted gene therapies that achieve long-term efficacy and enable patients to remain asymptomatic without continuous invasive treatment.
Initially, we are developing DNA therapeutics for patients with rare diseases of the central nervous system (CNS) that are caused by single-gene defects. Our lead programs are focused on Aromatic L-Amino Acid Decarboxylase Deficiency (AADC-D), Friedreich’s ataxia (FA) and Angelman syndrome (AS). AADC deficiency arises from mutations in the DDC gene and in its profound form results in severe developmental failures, global muscular hypotonia, severe long-lasting seizures, and premature death. FA is a rare inherited neurodegenerative disease that results in a physically debilitating, life-shortening condition. AS is a rare disorder characterized by a severe cognitive disability. Agilis Biotherapeutics is also pursuing development work in the rare CNS disease 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. The diseases we target usually appear during childhood, creating significant illness and long-term quality-of-life consequences, that often include premature mortality.
In meeting our primary responsibility to patients with rare diseases, we are committed to actively seeking and investing in best-in-class discoveries, knowledge, technology platforms, and strategic partnerships that will enable us to rapidly advance our DNA therapeutics. Our Friedreich’s ataxia program is enhanced by our worldwide exclusive strategic partnership with Intrexon Corporation, which provides access to Intrexon’s industrial-scale engineering platforms and technologies for the development of novel DNA therapeutics.
We have assembled a world-class body of knowledge and expertise to build a transformational DNA therapeutics company. The company is led by a team of executives and scientific advisors with diverse backgrounds and talents in the development of genetic medicine. These experts include leaders in the fields of synthetic biology, gene therapy, viral vectors, and gene transcription and regulation; and world-leading experts in rare diseases, drug delivery, systems biology, biomedical engineering, and molecular biology.
Agilis Biotherapeutics was founded by the Principals at Griffin Securities, Inc. and is supported by dedicated investors, including Third Security LLC, a venture capital fund led by Randall J. Kirk; Biomatrix Partners; Gold Bench Capital; and White Rock Capital; alongside other private investors. Together with the leadership team of Agilis Biotherapeutics, the group shares a passion to improve the therapeutic options for patients with rare genetic diseases.