Hereditary hearing loss is caused by a mutation in a single gene, and it impairs hearing from birth. For millions of people all over the world, it is a life-long disability that has no cure and no approved drugs for treatment. But a Boston-based biotech company called Akouos has begun developing a gene therapy that helps patients take back their hearing.
Akouos has raised $50 million in its first round of major funding to finance research and development for correcting genetic forms of hearing loss. It has received the first tranche of $25 million and wishes to demonstrate the effectiveness of the treatment in humans in the next five years. There are no specifics about the financing, but the remainder of the funding will presumably come with the success of the initial trials.
The recent round of funding was led by 5AM Ventures and New Enterprises Associates. These two companies previously invested $7.5 million in Akouos in November 2017. Previous investor Partners Innovation Fund, contributed, with RA Capital Management, Sofinnova Ventures, and Novartis Venture Fund also joining the investor pool.
Apart from the recent financing, Akouos announced the appointment of Michael McKenna, Akouos co-founder, as Chief Medical Officer. He has been a professor at Harvard Medical School since 2006. Akouos also appointed Jennifer Wellman as Senior Vice President. She was instrumental in the company’s regulatory work for gene therapy.
Hearing Loss in Detail
Hearing involves the inner ear’s hair cells translating auditory vibrations into electrical signals. The auditory nerve then transports these signals to the brain. There it is processed as sound. In the deaf, hair cells are present in their ears, but they do not function because of the genetic mutation. Akouos is using an engineered Adeno-associated virus (AAV) to send an operational copy of the mutated gene into the inner ear’s cells.
Akouos’ AAV approach is similar to Spark Therapeutics’ Luxturna, which received approval from the FDA for gene therapy last year. Luxturna treats a rare inherited type of blindness. Like the Spark Therapeutics gene therapy, Akouos’ therapy targets patients with a genetic disease. It is based on the research of the founder, Luk Vandenberghe, an ophthalmology professor at Harvard Medical School. He is also the director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear. Akouos’ CEO Manny Simons said the Vandenberghe method proves that the AAV can reach depths other viral vectors cannot.
Akouos is now testing its treatment on animals. By next year the company hopes the FDA will allow human testing, and to have clinical trials in three years. Simons remarked that Akouos is also using the fund to develop a wider range of therapies to treat hearing loss.
Hearing Aids An Industry on the Move
Apart from Akouos, there are other companies developing important treatments to address hearing loss treatment. One of them is Frequency Therapeutics from MIT, which focuses on activating the “progenitor” cells, or dormant cells that have the ability to transform into other cells. The company raised more than $30 million in funding in 2017with the goal of getting the progenitor cells to form new hair cells in the inner ear.
Another company is Decibel Therapeutics, which received close to $55 million in financing in June. It is developing a pipeline of experimental treatments, one of which includes gene therapy for hearing loss. While it has not detailed its gene therapy initiatives, Decibel Therapeutics aims for human testing in two years. Some of its treatments closer to clinical testing are meant to prevent other types of hearing loss.
But the company that has stayed ahead of the curve is Novartis, which is in early-stage clinical testing for gene therapy for hearing loss. Novartis has asserted that a gene dictates the growth of hair cells in the ears, but this growth ceases shortly after birth. Their gene therapy, CGF166, reintroduces the gene into the cochlea via a virus and turns the gene on like a switch. This stimulates the hair cells to grow and develop fully, priming the ears to function as normally as possible.
Changing Lives Through Hearing Loss Treatment
Gene therapy for the hearing-impaired is a new step toward helping people reclaim their senses, and experience the world normally. It is a welcome revolution. Soon, people will not be as dependent on hearing aids or implants anymore, instead of turning to gene therapy for a more permanent solution. The world can expect more breakthroughs as these bold companies make bold innovations to treat hearing loss in the years to come.