Articles from n-Lorem

n-Lorem, a nonprofit foundation that discovers, develops and provides personalized experimental antisense oligonucleotide (ASO) medicines to patients with nano-rare mutations (1-30 known worldwide) for free, for life, announced today the publication in Nucleic Acids Research [https://doi.org/10.1093/nar/gkag504] that details its experience with the first 329 nano-rare patient applications for treatment and a summary of the results to date in nano-rare patients treated with experimental ASOs medicines provided by n-Lorem.

n-Lorem, a nonprofit foundation, today announced that nine patients with an ultra-rare type of amyotrophic lateral sclerosis (ALS) have been treated during the first year of a multi-year grant from the National Institutes of Health (NIH). The work has been conducted as part of Silence ALS, an initiative that aligns Columbia University and n-Lorem on the joint mission to develop personalized experimental antisense oligonucleotide (ASO) medicines to treat ALS patients with ultra-rare, pathogenic gene mutations.

n-Lorem, a nonprofit foundation, and GondolaBio, a clinical-stage biotechnology company founded in 2024 and dedicated to developing novel medicines for patients living with genetic diseases, today announced that the two organizations have entered into multiple collaborations to discover novel antisense oligonucleotide (ASO) medicines for patients who are not currently served by available treatment options. n-Lorem will leverage its robust, automated, high-capacity ASO discovery platform and significant expertise in ASO technology to discover, screen and select ASO medicines to two prespecified biological targets in genetic diseases with significant unmet need. Once selected, GondolaBio will move forward in development of these new medicines.

n-Lorem, a nonprofit foundation, announced a new partnership with Hongene Biotech Corporation that supports n-Lorem’s efforts to discover and provide personalized experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life. As part of its commitment to n-Lorem and nano-rare patients, Hongene will provide amidites, compounds essential to developing ASO medicines, to n-Lorem to offset the costs of developing ASO medicines for up to 20 nano-rare patients.

n-Lorem, a nonprofit Foundation, has collaborated with Across Healthcare, a technology innovator, to develop a first-of-its-kind submission platform and system that allows research physicians from across the United States to submit applications for treatment for their patients with nano-rare illnesses. Robust data on each patient’s genotype and phenotype is required to enable the decision as to whether their mutation will become a drug target in n-Lorem’s discovery and development pipeline. As a non-profit, n-Lorem’s mission is to discover, develop and provide treatment for free, for life to the nano-rare patients that are selected after a thorough internal and external application review process. This process includes a physician application for their patient, evaluation of the disease-causing genetic mutation and patient’s clinical manifestations, and discussion with a team of external experts on the Access to Treatment Committee (ATTC) to make recommendations on acceptance.

n-Lorem, a nonprofit foundation, announced today that it has surpassed its 2022 fundraising goal of $8 million with more than $17 million in committed cash in 2022. As a non-profit, this support is critically important for n-Lorem’s mission to discover and develop personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

n-Lorem, a nonprofit foundation, today announced that Sarah Glass, Ph.D. has been promoted to Chief Operating Officer (COO). In this role, Dr. Glass will lead the day-to-day operations of n-Lorem and continue to implement strategic initiatives that ensure n-Lorem is positioned to meet the current and projected demand for personalized ASOs for nano-rare patients. n-Lorem’s mission is to discover, develop and provide individualized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients with the same mutation worldwide) for free, for life.

n-Lorem, a nonprofit foundation, today announced that The Wolverine Foundation has made a donation of $3 million to support n-Lorem’s efforts to discover and provide experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life. As part of the collaboration, The Wolverine Foundation and its assembled team of renowned scientists and researchers will work with n-Lorem to expedite research and ASO discovery for allele-specific gene targets associated with neurodevelopmental diseases. As the collaboration proceeds and research targets are achieved, n-Lorem could receive donations totaling up to $8.5 million to support the development of experimental ASO medicines.

n-Lorem, a nonprofit foundation, is collaborating with Cytiva, a global life sciences leader, 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.

n-Lorem a nonprofit Foundation, discovering and providing experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life, today announced that it has appointed John Maraganore, Ph.D., Chair of its Advisory Council. In this role, John Maraganore will chair a group of n-Lorem advisors with a focus on exploring fundraising opportunities, raising awareness of n-Lorem and its mission.