HOW DOES THIS TREATMENT WORK?
Inactive
AVB-202-TT Gene Therapy
Solid Biosciences
Inactive
AVB-202-TT is a novel gene therapy candidate being developed for the treatment of FA. AVB-202-TT, like other potential gene replacement treatments for FA, is intended to replace the frataxin gene across relevant tissues, with the goal of preventing progression or reversing the course of the disease.
HOW DOES A GENE THERAPY GET DEVELOPED?
INACTIVE: Stages of Development for Solid Biosciences’ AVB-202-TT Gene Therapy
The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.
Barry Byrne, M.D., Ph.D., and Manuela Corti, P.T., Ph.D., of University of Florida’s Powell Gene Therapy Center and the MDA Care Center at UF Health developed a gene therapy approach for FA. They founded AavantiBio with a private financing round includes a $15 million equity investment from Sarepta.
September 30, 2022: Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on therapies for Duchenne muscular dystrophy (Duchenne), and AavantiBio, Inc, announced that the companies entered into a definitive merger agreement whereby Solid acquired AavantiBio, including its pipeline assets and net cash. The combined company is focusing on advancing a portfolio of neuromuscular and cardiac programs, led by SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne. Additional pipeline programs include AVB-202, a gene transfer candidate for the treatment of Friedreich’s ataxia, AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional assets for the treatment of undisclosed cardiac diseases. Bo Cumbo, who was the Chief Executive Officer of AavantiBio, assumed the role of President and CEO of Solid Biosciences.
AVB-202-TT is in preclinical development and utilizes a dual route of administration (via both intrathecal and intravenous routes) to target disease pathology in brain and the heart. Preclinical data from three animal models, including mouse and nonhuman primate, supported preclinical proof of concept. Early findings in preclinical studies demonstrated improved survival and cardiac function, as well as mitochondrial function in mice.
