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On April 4, Akouos, inc. dedicated to the development of gene therapy based on adeno-related virus (AAVs), announced the completion of more than $100 million in Round B financing to advance its first gene therapy, AK-OTOF, into clinical development to treat sensory neurohearing loss caused by mutations in the otferrin (OTOF) gene.
cochlear implant is a spiral organ in the inner ear that contains 30,000 sensory cells that help your hearing.
each cell (called hair cells) has thousands of working proteins that allow us to convert nanoscale movements into nerve electrical signals.
the structure of these proteins is based on scripts provided by human DNA, changes in people's DNA can cause the protein to fail to function, which in turn affects hearing in many different ways.
tens of thousands of children are born with hearing difficulties every year.
about 50-60% of these cases are caused by changes in DNA.
to date, more than 150 genes are known to be directly related to deafness.
by comparing genetic data between deaf and normal hearing family members, scientists have been able to understand the location of these deaf-related genes.
by identifying these genes and understanding the protein function they encode, scientists began developing gene therapies designed to transfer DNA fragments that encode normal proteins to cells in the cochlea, enabling them to produce functional proteins and restore hearing.
Akouos (Photo: Akouos) To develop treatments for hearing loss and deafness, Akouos is linking scientific insights into the genetics of hearing loss to recent advances in gene therapy.
first, understand which genes are affected and how they damage hearing.
healthy copies of these genes can be loaded into engineering viral vectors, into the cells involved and produced functional proteins.
is currently conducting preclinical studies to support its initial clinical trial, which will assess the safety and effectiveness of gene therapy in patients with sensory neurosensory hearing loss due to mutations in the OTOF gene.
normal OTOF function activates auditory neurons by enabling sensory cells in the ear to respond to sound stimuli and release neurotransmitters.
functional OTOF is unable to transmit auditory signals to the brain.
AK-OTOF uses AAV vectors to transmit healthy copies of the OTOF gene to cochlear hair cells in order to restore long-term physiological hearing after a single administration.
"This is expected to be a year of rapid growth for Akouos, and we are pleased to be supported by world-class investors in Round B financing to advance our current project and the development of potential inherited hearing impairment drugs," said Dr. Manny Simons, founder, president and ceo of Akouos.
" References: 1Akouos Closes $105 Series B Finance B, Retrieved March 03,2020, from .2 Akouos Official Website Source: Round B Raises More Than $100 Million, Gene Therapy Newcomers Target Hearing Loss.