HOW DOES A GENE THERAPY GET DEVELOPED?

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.

Link to Solid Biosciences

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.

HOW DOES THIS TREATMENT WORK?

Gene Therapy for Friedreich’s Ataxia

Image of Video title - Gene Therapy

News and Press Releases

News
| Jul 16, 2024

FARA Advocates for Effective Inclusion of Children Early in Clinical Trials at Pediatric Inclusion Roundtable

Advocacy, FARA News
News
| Jul 16, 2024

2023 Donor Impact Report

FARA News
Blog
| Jul 15, 2024

Meet the Community – Caroline Spencer

Community
News
| Jul 15, 2024

LEXEO Therapeutics Announces Positive Interim Phase 1/2 Clinical Data of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

Industry News
Blog
| Jul 11, 2024

Join FA Teen Hangouts on July 13, 2024!

Ambassador, Hangouts
Blog
| Jul 8, 2024

Meet the Community 2: A Closer Look – Marieke van Driel

Community
Blog
| Jul 1, 2024

Meet the Community – Claire Juip

Community
Blog
| Jun 20, 2024

2024 Cure FA Soirée Recap

Events
Blog
| Jun 20, 2024

Jackson Walters

Memorial
News
| Jun 18, 2024

C-Path Partners with FARA to Fortify RDCA-DAP and Further Accelerate Drug Development with new Friedreich’s Ataxia Data

Partner News
Blog
| Jun 17, 2024

Meet the Community – Kyle Duhon

Community
Blog
| Jun 10, 2024

Meet the Community – Hayden Clay Rogers

Community