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N-Lorem, Hongene Biotech Partner on ASO Therapies for Nano-Rare Diseases

NEW YORK – Nonprofit N-Lorem has partnered with Hongene Biotech to develop antisense oligonucleotide (ASO) therapies for extremely rare diseases.

Under the agreement announced Wednesday, Hongene, a Union City, California-based company specializing in nucleic acid manufacturing, will provide N-Lorem with synthetic oligonucleotide building blocks, called amidites, to offset the costs of ASO development for up to 20 "nano-rare" patients.

The companies did not disclose financial details of the collaboration.

"In many cases, nano-rare patients will succumb to their disease without ever having hope of a treatment that will halt their progressive diseases," Stan Crooke, chairman, founder, and CEO of N-Lorem, said in a statement. "At N-Lorem … our approach enables us to discover and develop an individualized ASO medicine that targets a nano-rare patient's unique mutation, and we do this for free, for life."

San Diego-based N-Lorem works with numerous researchers and organizations to develop ASO therapies for genetic conditions that afflict between one to dozens of individuals worldwide.

"The need from the nano-rare community is substantial, and we cannot do this alone," Crooke said. "Thanks to the generosity of Hongene, we will be able to discover and evaluate ASO medicines for nano-rare patients without having to worry about sourcing crucial amidites."