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On October 18, Eli Lilly announced the acquisition of gene therapy company Akouos, Inc.
(NASDAQ: AKUS) for $610 million, another important expansion
in the gene therapy field following Lilly's acquisition of Prevail Therapeutics for $1.
04 billion in 2020.
Akouos, Inc.
(NASDAQ: AKUS) is a gene therapy company founded in 2016, headquartered in Boston, USA, co-founded by experts in neurology, genetics, inner ear drug delivery and AAV gene therapy, Akou means
"listen" in Greek.
The company is developing a family of adeno-associated virus (AAV) vector-based gene therapies primarily for the treatment of sensorineural hearing loss
.
Akoouos U.
S.
stocks soared nearly 90% premarket, and Akoouos has the Ancestral AAV (AAVAnc) technology platform
developed by Dr.
Luk Vandenberghe, a professor of ophthalmology at Harvard Medical School.
Its first genetic product, AK-OTOF, is currently in clinical development to treat sensorineural hearing loss caused by mutations in the ototofin otoferlin (OTOF) gene, and the company's other pipeline includes a variety of inner ear diseases
.
Akouos' overall pipeline (Source: Akouos official website)
At present, most of the treatment of deafness is the use of physical methods such as hearing aids and cochlear implants, and there are no FDA-approved drugs
so far.
AK-OTOF uses an AAV vector to deliver the OTOF gene to damaged cochlear cells and express a normally functioning OTOF protein to treat patients with sensorineural deafness due to mutations in the OTOF gene, who often have severe bilateral deafness
from birth.
About 200,000 people worldwide have inherited hearing loss
due to OTOF gene mutations.
Luk Vandenberghe (Credit: Xconomy)
Compared to other viral vectors currently under development, the AAVAnc vector platform has the potential to overcome innate immunity in order to address diseases that target an unmet need for a
broader patient population.
Among them, Anc80 is the putative ancestor of AAV1, 2, 3, 6, 7, 8, 9 and rh.
10 and is also considered the most advanced gene therapy vector in the 40,000 novel AAVAnc combinations of this technology platform, and Selecta Biosciences has previously been authorized for specific indications, including methylmalonic acidemia
.
In addition, Anc80L65 has been shown to efficiently transform sensory cells in the inner ear and to cochlear cell types
previously thought to be out of reach for AAV.
Original English text:
