Since there is no causal medical treatment for sensorineural hearing loss today, the goal of ACOUSIA is to establish drug development of small molecule compounds that trigger regeneration of sensory hair cells and allow for causal treatment of sensorineural hearing loss in patients who already lost their hair cells years ago.
Acousia’s approach is based on the fact that small molecule compounds have increasingly been used to dedifferentiate and reprogram differentiated somatic cells in order to enhance or substitute genetic reprogramming approaches (Anastasia et al, 2010). Taken to the situation of hair cell regeneration in the inner ear, a small molecule based dedifferentiation/reprogramming concept, targets the differentiated supporting cells that are retained in the sensory epithelium after hair cell loss.
During their research the scientific founders, Prof. Löwenheim and Prof. Wiesmüller, identified and patented a small molecule – otopotin – that dedifferentiates otic supporting cells into otic progenitor-like cells that proliferate and redifferentiate into hair cell and supporting cell in vitro. This compound has been selected as a hit compound from a small molecule library by screening primary supporting cells derived from adult mouse cochlea organ culture. Validation of the compound in the tissue context has been achieved in an organ culture assay. Otopotin and other related derivative compounds have been patent protected and are available for further development. Based on this proof of concept further efforts in a hit-to-lead and consecutive lead optimization (LO) drug development program will be employed.
- Anastasia L, Pelissero G, Venerando B, Tettamanti G (2010) Cell reprogramming: expectations and challenges for chemistry in stem cell biology and regenerative medicine. Cell death and differentiation 17:1230-1237.