Akouos Announces New Data at the American Society of Gene & Cell Therapy 2019 Annual Meeting

Non-human primate data demonstrate potential of the Akouos gene
therapy platform to develop precision medicines for inner ear disorders

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

“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.”

Poster Presentation

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

Session Date and Time: April 29, 2019, 5:00 – 6:00 p.m. ET

Abstract is available on the ASGCT Meeting website.

About Akouos

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.


Katie Engleman, 1AB