Participated · Series B · Mar 2020

Akouos develops precision gene therapies for sensorineural hearing loss using an adeno-associated viral (AAV) vector-based platform. Its lead program, AK-OTOF, targets hearing loss caused by mutations in the otoferlin (OTOF) gene. The company plans to advance AK-OTOF to first-in-human clinical studies subject to IND filing and acceptance. Proceeds from its recent financing will also support establishing in-house GMP manufacturing at new offices in the Boston Seaport and accelerating multiple pipeline programs with well-defined mechanisms and gene targets. Akouos intends to expand its teams across research, clinical development, and manufacturing. The company has strategic partnerships with Massachusetts Eye and Ear and Lonza, Inc. Akouos is a precision genetic medicine company focused on developing targeted adeno-associated viral (AAV)-based gene therapies for sensorineural hearing loss, with an initial emphasis on monogenic forms that cause profound deafness. The company leverages a proprietary AAV gene therapy platform and has strategic partnerships with Massachusetts Eye and Ear and Lonza. Proceeds from a recent $50 million Series A will support advancement of its lead program to first-in-human clinical studies and accelerate multiple pipeline programs addressing other validated gene targets. Akouos has assembled clinical and scientific leadership, expanded its board, and formed a scientific advisory board composed of world-leading experts in gene therapy and hearing loss. The company states its approach aims to deliver one-time precision therapies to treat underlying genetic causes of hearing disorders. Akouos develops gene therapies aimed at restoring and preserving hearing and balance using in silico-designed Anc-AAV viral vectors. The company entered strategic license agreements with Lonza and Massachusetts Eye and Ear to obtain exclusive rights to the Anc-AAV gene therapy platform and broad sublicensing rights. Anc-AAVs were first developed in the laboratory of Dr. Luk H. Vandenberghe and are intended for application across hearing and balance disorders. Akouos was launched in 2017 by Dr. Manny Simons, who serves as CEO; its scientific founders and advisors include Drs. Michael McKenna, William Sewell, Richard Smith and Luk Vandenberghe. In conjunction with closing a $7.5M seed financing, the licensing and partnership framework is designed to accelerate AAV gene therapy development for hearing and balance conditions. Dr. Vandenberghe is a listed inventor on Anc-AAV intellectual property licensed to Lonza and Akouos and receives royalties.