Parexel and n-Lorem Foundation Partner to Support Development of New Investigational Therapies for Nano-rare Patients

BOSTON & DURHAM, N.C.--(BUSINESS WIRE)-- Parexel, a leading global clinical research organization (CRO), and n-Lorem, a nonprofit foundation, today 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 (1 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.

“This collaboration exemplifies Parexel’s commitment to nano-rare patients, who present insurmountable challenges to the current healthcare systems. Together with our partners and generous donors, n-Lorem is addressing these challenges one nano-rare patient at a time,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Currently, n-Lorem has over 50 nano-rare patients and each patient requires both an individual natural history study and a clinical study. We are pleased to be partnering with Parexel, an organization with extensive rare disease expertise that is an expert in managing clinical trials. Parexel will provide the necessary systems to assure that each patient’s natural history and clinical studies are well managed, which will be very important as we accept more nano-rare patients and collect data that informs our future decisions and aggregate experience.”

“n-Lorem and Parexel demonstrate a shared passion and commitment to making a difference for nano-rare patients,” said Jamie Macdonald, CEO at Parexel. “We are pleased to partner with n-Lorem on this work and to contribute our collective global experience and resources to support their efforts.”

“Drug development for rare diseases is complex and presents many challenges,” added Clare Grace, Ph.D. Chief Patient Officer at Parexel. “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.”

“Parexel has deep roots in supporting the nano-rare patient community with their in depth understanding of the challenges and nuances of rare disease research. Parexel’s resources and guidance from their team of leading medical, regulatory and real-world data experts will translate into more robust and supportive clinical research at n-Lorem,” said Sarah Glass, Ph.D., Chief Development Officer, n-Lorem Foundation. “n-Lorem is a very nimble organization limited only in our funding and resources. Partnering with Parexel will allow us to expand our clinical research infrastructure, broadening access to n-Lorem for nano-rare patients.”

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

About Parexel

One of the largest clinical research organizations, Parexel supports the development of innovative new medicines to improve the health of patients. We provide services to help life sciences and biopharmaceutical customers everywhere transform scientific discoveries into new treatments. From decentralized clinical trials to regulatory consulting services to leveraging real-world insights, our therapeutic, technical, and functional ability is underpinned by a deep conviction in what we do. For more information, visit parexel.com and follow us on LinkedIn, Twitter, and Instagram.

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 50 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn and YouTube.

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