Non-human primate data demonstrate potential of the Akouos gene therapy platform to develop precision medicines for inner ear disorders.
Boston, Mass. – April 15, 2019 – Akouos, a precision genetic medicine company developing gene therapies to restore and preserve hearing, announced today that data from its inner ear gene therapy platform will be presented during the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), being held April 29 – May 2, 2019 in Washington D.C.
The data demonstrate dose-dependent transduction of multiple cochlear cell types and preliminary safety of Anc80AAV, a rationally-designed synthetic adeno-associated viral (AAV) vector, in non-human primates. These data support intracochlear administration of Anc80AAV as a promising strategy to treat a wide range of hearing and balance disorders.
“Akouos is building the leading gene therapy platform for inner ear disorders, and we are focused on translating our technology into medicines that could ultimately improve the lives of millions of individuals and families,” said Manny Simons, Ph.D., founder, president and CEO of Akouos. “We are pleased to present these data at ASGCT as an early demonstration of our platform’s potential to address many forms of hearing loss with our precision therapies.”
Title: The Adeno-Associated Viral Anc80 Vector Efficiently Transduces Hair Cells in Cynomolgus Macaques (M. fascicularis): Development of a Non-Human Primate (NHP) Model for Cochlear Gene Therapy
Authors: Shimon Francis, Michael McKenna, Yuan Gao, Robert Ng, Enping Qu, Luk Vandenberghe, William Sewell, Emmanuel Simons, Michelle Valero
Session Date and Time: April 29, 2019, 5:00 – 6:00 p.m. ET
Abstract is available on the ASGCT Meeting website.
Akouos is a precision genetic medicine company dedicated to developing gene therapies that restore and preserve hearing. Leveraging its adeno-associated viral (AAV) vector-based gene therapy platform, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, the Company was founded in 2016 by world leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy. Akouos has strategic partnerships with Massachusetts Eye and Ear Infirmary and Lonza, Inc. For more information, please visit www.akouos.com.
About Anc-AAV Technology
Ancestral AAV (Anc-AAV) technology, developed in the laboratory of Luk Vandenberghe, uses computational and evolutionary methods to predict novel conformations of the adeno-associated viral particle. Anc80AAV, a predicted ancestor of AAV1, 2, 3, 6, 7, 8, rh.10, and AAV9, has demonstrated preliminary safety and effective gene delivery in both mice and non-human primates in numerous preclinical studies.