More than half of all hearing loss cases are hereditary. Myelin protein zero-like 2, encoded by MPZL2, is widely expressed in cochlear cells in the inner ear. Mutations in MPZL2 have been identified as the second most prevalent cause of mild to moderate hereditary hearing loss.

It has been previously demonstrated that intranasal treatment with COG-201, an AAV9-shRNA designed to target the down-regulation of the 5-HT2A receptor, significantly decreased anxiety and improved memory in mice and rats.

In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, GENE-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids.

Genespire Srl has closed a €46.6 million (~$52 million) series B financing to support its work developing off-the-shelf gene therapies based on immune shielded lentiviral vectors (ISLVs) for pediatric patients with genetic diseases.