Akouos raises $50M to take hearing loss gene therapy into humans

Akouos has raised $50 million to take a gene therapy treatment for hearing loss into the clinic. The Boston-based biotech unveiled the series A round alongside the appointment of a Spark Therapeutics co-founder and other organizational changes.

5AM Ventures and New Enterprise Associates (NEA) got Akouos off the ground last year with a $7.5 million seed round that equipped the startup to license an ancestral AAV gene therapy platform from Lonza and Massachusetts Eye and Ear. Akouos aims to use the technology to create therapies capable of efficiently delivering genes to sensory cells found on a sensitive epithelial membrane in the inner ear, and thereby treat certain forms of hearing loss.

The team is initially targeting monogenic forms of sensorineural hearing loss. These forms arise when changes to single genes cause sensory cells or nerve fibers in the inner ear to malfunction. That focus gives Akouos plenty of targets to choose from. Researchers have implicated tens of genes in hearing loss, although some—notably GJB2—affect more people than others. A study by Akouos’ co-founder Richard Smith, M.D., implicated 49 genes in hearing loss. GJB2 accounted for one-fifth of cases.

Akouos is now set to build on this research and the efforts of its other scientific founders—Michael. McKenna, M.D., William Sewell, Ph.D. and Luk H. Vandenberghe, Ph.D.—using $50 million in series A funding.

5AM and NEA co-led the round with the support of fellow seed round backer Partners Innovation Fund and a trio of new, big name investors: Sofinnova Ventures, RA Capital Management and Novartis Venture Fund.

Akouos will use the series A cash to advance a lead candidate addressing an as-yet-undisclosed genetic fault into first-in-human trials. In parallel, the biotech will work on multiple other programs to expand the potential benefits of its gene therapy platform to patients whose hearing loss has different genetic roots. 

Other researchers are also going after hearing loss. Novartis is four years into a trial of a gene therapy treatment for hearing loss caused by environmental factors, while multiple preclinical teams are exploring fixes for genes including ATOH1 and TMC1.

Akouos enters this competitive environment with a strong hand. The biotech began life with the support of experts in fields including inner ear drug delivery and AAV gene therapy, and has since added to its team and board. The changes include the appointment of Jennifer Wellman, a co-founder and former head of product development strategy at Spark, as SVP of regulatory.

Kush Parmar, M.D., Ph.D., managing partner at 5AM, thinks Akouos’ team gives it an edge

“Akouos has assembled the world’s leaders in neurotology, genetics, inner ear drug delivery and gene therapy under one roof to develop gene therapies that aim to improve hearing with a one-time administration. This team, focused on highly validated biology and targeted delivery, and leveraging a state-of-the-art proprietary platform technology, is uniquely positioned to change the standard of care for hearing disorders,” Parmar said in a statement.