RARE Daily

n-Lorem Foundation Introduces First ‘Partner in Excellence’ to Support and Accelerate the Treatment of Nano-rare Patients

June 15, 2022

n-Lorem, the nonprofit ASO developer for ultra-rare diseases with 30 or fewer patients, named its first ‘Partner in Excellence’ collaboration with Columbia University Irving Medical Center.

The program at Columbia will be led by co-principal investigators Wendy Chung and Neil Shneider, leading research physicians treating patients with extremely rare genetic mutations. n-Lorem said its 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.

“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 Crooke, founder, CEO, and chairman of n-Lorem Foundation.”

n-Lorem’s Partner in Excellence collaborators provide 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 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.

“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,” said Wendy Chung, director of DISCOVER and the Kennedy Family Professor of Pediatrics at Columbia University Vagelos College of Physicians and Surgeons. “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.”

Photo: Wendy Chung, director of DISCOVER and the Kennedy Family Professor of Pediatrics at Columbia University Vagelos College of Physicians and Surgeons

Author: Rare Daily Staff

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