Benzinga | Jan 18, 2022 09:07AM EST

n-Lorem Foundation Reports Partnership With Alnylam To Expand Technology Access To RNAi Therapeutics For Patients With Nano-Rare Diseases, No Terms Disclosed

Alnylam's RNAi expertise and financial donation bolster efforts to reach more nano-rare patients where a RNAi targeted approach would be most appropriate

SAN DIEGO--(BUSINESS WIRE)-- n-Lorem, a nonprofit Foundation, discovering and providing experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life, today announced a new partnership with Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY).

"Discovering, developing and providing individualized medicines for nano-rare patients is a monumental task. At n-Lorem, using ASO technology, we take advantage of three decades of experience, unparalleled quality control and integrated safety databases that are used to select frequency and dosage for each individual ASO therapy. As we expand our highly selective and versatile model to other amendable technologies, it is important for us to maintain a high level of quality," said Stanley T. Crooke, M.D., Ph.D., Founder, Chief Executive Officer and Chairman, n-Lorem Foundation. "Alnylam's RNAi therapeutic technology is the perfect complement to Ionis' ASO technology, both are well understood, proven drug discovery technologies. Adding Alnylam as a partner is another step in the creation of a broad network of partner stakeholders committed to treating nano-rare patients."

"We are pleased to partner with the n-Lorem Foundation in identifying opportunities to bring our novel RNAi technology platform to potentially help patients with nano-rare diseases. At Alnylam, we are committed to identifying pathways to expand access to our medicines and we applaud the work of the foundation in bringing together industry partners who are driven to provide options for patients that have little or no options for treatment," said Akshay Vaishnaw, M.D., Ph.D., President at Alnylam.

"Although we have the tools and expertise to understand nano-rare patients at a genetic level, the extreme rarity of these patients present insurmountable challenges to the current healthcare systems," said Dr. Sarah Glass, Chief Development Officer, n-Lorem Foundation. "n-Lorem is addressing these challenges one nano-rare patient at a time and is supported through a consortium of well-renowned and diverse organizations worldwide. These include our biotechnology and pharmaceutical partners, including Ionis Pharmaceuticals, Biogen, Ultragenyx and today Alnylam; our clinical research partners, including Charles River Labs, Korea Institute of Toxicology and Parexel; our contract manufacturing partners, including ChemGenes, Cytiva and Nitto Avecia; and many other collaborations that make n-Lorem's mission possible."

Learn more about n-Lorem's mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

* Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease

* Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time