Columbia University Irving Medical Center joins n-Lorem to facilitate ASO treatment for advance understanding of and improve healthcare for nano-rare patients

n-Lorem, a nonprofit foundation, today announced the first ‘Partner in Excellence’ collaboration with Columbia University Irving Medical Center. The program at Columbia will be led by co-principal investigators Wendy Chung, M.D., Ph.D., and Neil Shneider, M.D., Ph.D., leading research physicians treating patients with extremely rare genetic mutations.

Partnering with world-class institutions with clinical and research expertise in rare diseases is a critical component of n-Lorem’s consortium approach to treating nano-rare patients. n-Lorem is the only non-profit foundation using a robust and proven ASO technology to discover and provide experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life.

“Nano-rare patients have genetic diseases that often are unique to a single individual in the world. In the past, there was little that could be done for these patients, but today, n-Lorem and our Partner in Excellence investigators are able to meet the needs of some of these nano-rare patients, one patient at a time,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “I am pleased to announce our first Partner in Excellence collaborators and to confirm our support in working closely with Drs. Chung and Shneider to develop ASO therapies for some of their patients.”

n-Lorem’s Partner in Excellence program aligns research physicians, world-class institutions and n-Lorem with the common goal to provide the highest quality healthcare and treatment for nano-rare patients. By taking an individualized approach to each nano-rare patient, n-Lorem is able to develop an optimal ASO medicine for that patient. n-Lorem’s Partner in Excellence collaborators are integral to this approach, providing genetic and phenotypic characterization, assessing the viability of ASO technology for their patients, submitting proposal-to-treat applications to n-Lorem, collecting critical natural history of each patient while n-Lorem develops a treatment, submitting an investigator-initiated application and administering the patient’s treatment. Through this program, n-Lorem provides support at every point in the process and works closely with principal investigators at partnering institutions as the experimental ASO therapeutic is being developed. As data is collected, each Partner in Excellence is encouraged to publish results to inform the community, highlight the pioneering work done at the institution and most importantly, enable others to apply lessons learned to expedite future programs for nano-rare patients.

n-Lorem’s recently announced Silence ALS Initiative with Columbia University exemplifies the power of a Partner in Excellence strategy where together, n-Lorem and Dr. Shneider are establishing an infrastructure to enable ASO discovery and development for carriers of nano-rare ALS gene mutations. Through the creation of a disease-focused initiative, n-Lorem can identify commonalities and introduce efficiencies in the ASO discovery and development programs.

“I have been working with n-Lorem since its inception and I have seen first-hand the measure of hope n-Lorem can bring to patients and families whose lives are threatened by disease. I am hopeful that as we continue to identify more patients who might benefit from this approach, we can expedite their path to treatment together,” said Neil Shneider, M.D., Ph.D, director of the Eleanor and Lou Gehrig ALS Center and the Claire Tow Associate Professor of Motor Neuron Disorders in the Department of Neurology at Columbia University Vagelos College of Physicians and Surgeons.

“For many of my nano-rare patients, we have little to offer in terms of clinical care. This is especially devastating when dealing with infants and children. I am hopeful that the work n-Lorem and I are doing together will translate into benefit for some of my patients today and in the future as we identify more patients who might be good candidates for n-Lorem’s approach,” said Wendy Chung, M.D., Ph.D., director of DISCOVER and the Kennedy Family Professor of Pediatrics at Columbia University Vagelos College of Physicians and Surgeons.

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

• Subscribe to n-Lorem’s Patient Empowerment Program, a podcast series for nano-rare patients
• Watch: n-Lorem Foundation: Offering Hope and Help to Nano-rare Patients
• Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time

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 140 applications for treatment with more than 60 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, YouTube and Instagram.

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