The companies will utilize AAVnerGene's tissue-specific, highly-transductive, and expressive new AAV (ATHENA) screening platform to efficiently select the best AAV vector for each cell type in a high-throughput manner. The company's platform could overcome limitations of earlier generation AAV vectors by achieving sufficient therapeutic expression of the transferred gene in a low dose through intravitreal injection.

"AAVnerGene's proprietary technology may create capsid libraries derived from artificial intelligence machine learning, DNA shuffling or directed evolution allowing a significant increase in AAV genetic payload capacity, production, and transduction with the ability to penetrate through the inner limiting membrane of the retina, thus potentially enhance the overall transduction efficiency of capsid library-derived AAV vectors," said Alvin Luk, PhD, CEO of Neurophth.

Under the agreement, Neurophth will make an initial cash payment of an undisclosed amount to AAVnerGene to test 100 AAV capsids. Within six-12 months, Neurophth will complete preclinical studies. After this, both companies will jointly create the capsids for further development. Upon selection of capsids, Neurophth wll pay AAVnerGene additional cash and/or potentially purchase AAVnerGene's common stock. Neurophth will pay AAVnerGene royalties on sales of any resulting commercialized gene therapy.

Neurophth's gene therapy granted orphan drug designation
Neurophth Therapeutics' lead candidate, NR082, an adeno-associated virus-based gene therapy, has been granted orphan drug designation by the U.S. Food...