n-Lorem Foundation and Harvard Medical School’s International Center for Genetic Disease Partner in Strategic Collaboration to Advance Therapies for Nano-Rare Diseases
The n-Lorem Foundation and the Harvard Medical School International Center for Genetic Disease (HMS-iCGD) have joined efforts to explore a collaborative framework aimed at advancing personalized treatments for individuals living with nano-rare genetic diseases. The collaboration began in September of 2024, and reflects a shared commitment to translational research, capacity building, and equitable access to innovative therapies.
Through this collaboration, the organizations intend to align efforts around the Global Initiative for Nano-Rare Therapy (GlNaRT), a joint initiative designed to support the discovery, development, and clinical application of individualized antisense oligonucleotide (ASO) therapies for patients with high unmet medical needs. GlNaRT brings together n-Lorem’s mission to provide individualized ASO medicines free for life with HMS-iCGD’s global leadership in genomic medicine, research, and international partnerships.
The scope of the
collaboration includes potential activities across translational research,
clinical services, and workforce development. These may encompass ASO discovery
and development, genetic research and data collection, training programs for
healthcare professionals, and the establishment of clinical and operational
infrastructure to support ASO therapies. The collaboration also envisions
jointly sponsored scientific and educational events, as well as engagement with
governments and global partners to help establish clinical treatment centers.
“This collaboration reflects a shared vision to accelerate the development of personalized therapies for patients who currently have no treatment options,” said representatives of the participating organizations. “By combining scientific expertise, clinical insight, and a commitment to global capacity building, GlNaRT aims to help transform care for individuals affected by nano-rare genetic diseases.”
About the n-Lorem Foundation
The n-Lorem Foundation is a non-profit organization dedicated to discovering, developing, and providing individualized antisense oligonucleotide therapies for patients with nano-rare genetic diseases—at no cost to the patient, for life. n-Lorem works at the intersection of science, medicine, and patient advocacy to address conditions that are often overlooked due to their extreme rarity.
Additional information about
the n-Lorem Foundation can be found here:
·
N-Lorem Foundation Home Page
Featured international
work from n-Lorem:
· N-Lorem Foundation Bringing Real Hope to ‘N-Of-1’ Patients
· ASOs Drive Foundation’s Work to Develop Treatments for the World’s Rarest Diseases
Upcoming Events by n-Lorem: https://www.nlorem.org/media/events/
Administrative leadership for the collaboration will be coordinated by Alireza Haghighi, MD, PhD, Director of the Harvard Medical School International Center for Genetic Disease, and Sarah Glass, PhD, Chief Operating Officer of the n-Lorem Foundation.
Together, n-Lorem Foundation
and HMS-iCGD aim to foster innovation, strengthen global expertise, and move
the field of personalized genomic medicine forward—bringing hope to patients
and families affected by the world’s rarest genetic conditions.