Parexel and n-Lorem yesterday announced a new partnership in support of n-Lorem’s effort to discover and provide experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (one to 30 patients worldwide) for free, for life. Parexel brings significant expertise to the partnership, having managed approximately 400 rare disease clinical studies over the past five years that contributed to 17 FDA drug approvals, the companies notified via a joint statement.
Stanley T Crooke, MD, PhD, Founder, CEO and Chairman, n-Lorem Foundation, said in the statement, “Currently, n-Lorem has more than 50 nano-rare patients and each patient requires both an individual natural history study and a clinical study……Parexel will provide the necessary systems to assure that each patient’s natural history and clinical studies are well managed, which will be important as we accept more nano-rare patients and collect data that informs our future decisions and aggregate experience.”
“Drug development for rare diseases is complex and presents many challenges,” said Clare Grace, PhD, Chief Patient Officer, Parexel, in the statement.
Grace added, “We have a unique opportunity with n-Lorem to help accelerate novel therapeutics for these patients with no other proven treatment options through our extensive scientific, clinical and regulatory rare disease expertise.”