Akouos Closes $105 Million Financing for Hearing Loss Gene Therapies

Akouos has raised $105 million in an oversubscribed series B financing to develop gene therapies to restore, improve, or preserve hearing.

Photo: Manny Simons, founder, president and CEO of Akouos

Pivotal bioVenture Partners led the round with participation from new investors Cormorant Asset Management, Cowen Healthcare Investments, EcoR1 Capital, Fidelity Management & Research Company, Polaris Founders Fund, Pagsgroup, Surveyor Capital, Wu Capital, and other institutional investors. Existing investors, 5AM Ventures, New Enterprise Associates, Novartis Venture Fund, Partners Innovation Fund, RA Capital Management and Sofinnova Investments also participated in the round.

In conjunction with the financing, the company appointed Vicki Sato and Heather Preston to its board of directors. During an illustrious career spanning more than 30 years, Sato, most recently a professor of management and also of molecular and cell biology at Harvard University, served as vice president of research at Biogen and later as president of Vertex. Preston, a medical doctor, has a background in venture capital, most recently at Pivotal bioVenture Partners, and before that at TPG Biotech, New Enterprise Associates, and JP Morgan Partners.

“These milestones bring us closer to potentially providing new options to the deaf and hard-of-hearing community, who have limited therapeutic options today,” said Manny Simons, founder, president and CEO of Akouos.

Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. The company’s first gene therapy development program, AK-OTOF, is for individuals with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene, a major cause of genetic hearing loss that affects an estimated 200,000 individual worldwide.

Normal otoferlin function enables the sensory cells of the ear (hair cells) to release neurotransmitter in response to stimulation by sound to activate auditory neurons. Without functional otoferlin protein, auditory signals received by the ear cannot be transmitted to the brain. AK-OTOF uses an adeno-associated viral (AAV) vector to deliver a healthy copy of the OTOF gene to cochlear hair cells, with the goal of restoring long-term physiologic hearing following a single administration to the inner ear. AK-OTOF is intended to treat individuals with sensorineural hearing loss due to mutations in the OTOF gene, who typically have severe bilateral hearing loss from birth, by promoting the expression of normal, functional otoferlin protein in affected cells of the cochlea.

Proceeds from the financing will be used to advance AK-OTOF to first-in-human clinical studies, establish the company’s in-house GMP manufacturing capabilities at its new offices in the Boston Seaport, accelerate the development of multiple pipeline programs that address other forms of sensorineural hearing loss with well-defined mechanisms and/or gene targets, and expand its team across research, clinical development and manufacturing.

Author: Rare Daily Staff