Articles from n-Lorem Foundation

n-Lorem, a nonprofit foundation, announced today that it will hold its third annual Nano-rare Patient Colloquium in Cambridge, Mass., where n-Lorem leaders and experts in the field will share updates on research being conducted to find treatments for patients with nano-rare diseases. The Colloquium, which is being hosted Oct. 20-21 by Biogen, will include >40 panelists and presenters, a scientific poster session, reception and an art auction (bidding is happening now) with more than 480 registrations to date.

n-Lorem, a nonprofit foundation, announced today the publication of new research characterizing a nano-rare mutation in a gene that is pivotal to normal cell signaling and cellular homeostasis. The mitogen-activated protein kinase 8 interacting protein 3 (MAPK8IP3) gene encodes for c-Jun N-terminal kinase-interacting (JNK-interacting) protein 3 (JIP3). Mutations in the MAPK8IP3 gene have been linked to severe neurodevelopmental disorders, however, the mechanisms of JIP3 underlying these deleterious effects were poorly understood. In this publication, n-Lorem scientists identified several significant roles JIP3 could play in the body, characterized a mutation in MAPK8IP3 and identified a path forward to use antisense oligonucleotide (ASO) medicines to address mutations in the MAPK8IP3 gene. The study, authored by n-Lorem scientist Wei Zhang, et al was published today in JCI Insights (JCI Insight. 2025;10(8):e187199).

n-Lorem, a nonprofit foundation, announced that Nucleic Acids Research has published its research that both characterized a pathogenic intronic splice variant of the glycogen branching enzyme (GBE1) gene and identified a splice-modulating antisense oligonucleotide (ASO) capable of restoring functional GBE1 protein in APBD patient cells. These data support a novel potential ASO treatment for nano-rare patients with same GBE1 pathogenic intronic splice variant. The findings in the paper, titled “Splice-modulating antisense oligonucleotides targeting a pathogenic intronic variant in Adult Polyglucosan Body Disease correct mis-splicing and restore enzyme activity in patient cells,” could offer, for the first time, a targeted therapy to restore gene function. The study, authored by n-Lorem scientist R. Thomas et al, was published in Nucleic Acids Research (NAR. 2025;53(13):gkaf658. https://doi.org/10.1093/nar/gkaf658).

n-Lorem, a nonprofit foundation, is celebrating five years of success in establishing a unique non-profit model bringing personalized experimental ASO medicines to nano-rare patients for free, for life. Having treated more than 30 nano-rare patients, n-Lorem has demonstrated that a non-profit model in which patients are accepted irrespective of their financial means is feasible. n-Lorem has shown that the efficiency of ASO technology, coupled to the unique FDA guidance documents for nano-rare patients, can rapidly bring effective treatments to even the most advanced patients, with an excellent safety and tolerability profile, and profound clinical benefit observed in many patients.

n-Lorem, a nonprofit foundation, drew more than 750 researchers, scientists, healthcare providers, patients and their families to its second annual Nano-rare Patient Colloquium hosted by Biogen. The two-day event in Cambridge, MA, united supporters of n-Lorem's mission, amplified the voices of nano-rare patients, and informed the community on the remarkable observations in n-Lorem treated nano-rare patients. To date, treatments have been initiated for more than 20 patients, with an additional patients expected to begin treatment by year’s end.

n-Lorem, a nonprofit foundation, will assemble patients, physicians, partners and supporters today and tomorrow, Oct. 30 & 31, 2024, for its second annual Nano-rare Patient Colloquium. In this public meeting, n-Lorem will provide valuable information to the nano-rare community, bring together those who support n-Lorem’s mission of discovering optimal treatments for nano-rare patients, give nano-rare patients a voice and highlight the industrialized processes n-Lorem has implemented to discover, develop and ultimately treat nano-rare patients with an optimized ASO for that individual patient. The meeting is being held at the Cambridge Marriott and includes more than 40 panelists and presenters and approximately 500 in-person and virtual attendees, all of whom are dedicated to finding treatment for nano-rare patients. Live streaming of the event can be accessed by visiting n-Lorem’s website: www.nlorem.org.

n-Lorem, a nonprofit foundation, announced today the publication of an article in the journal Nucleic Acid Therapeutics that explores potential regulatory approaches that could be taken to develop treatments for patients with rare heterozygous toxic gain-of-function (TGOF) mutations. The paper, titled Addressing the Challenges of Treating Patients with Heterozygous Gain of Function Mutations, was authored by Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO of the n-Lorem Foundation. (Crooke, S.T.,Nucleic Acid Ther, (2024) doi: 10.1089/nat.2024.0060)

n-Lorem, a nonprofit foundation, announced today the publication of a case study detailing the treatment and initial outcomes for a nano-rare patient who is receiving treatment with a personalized experimental ASO medicine discovered and developed specifically for that patient. In this report, most outcome measures including seizure severity, number of falls, overall speech and quality of life improved after treatment. The study, authored by the patient’s doctors Wendy K. Chung, M.D., Ph.D., of Boston Children’s Hospital, and Jennifer M. Bain, M.D., Ph.D. of Columbia University Irving Medical Center, along with n-Lorem, was published today in Nature Medicine (Zeigler, A., et al. Nature Medicine advanced online publication, Aug 2024).

n-Lorem, a nonprofit foundation discovering, developing and providing experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life, today announced that Andrew W. Lo, Ph.D., has joined its Board of Directors.

n-Lorem, a nonprofit foundation, discovering, developing and providing experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life, today announced that Alan Lotvin, M.D., has joined its Board of Directors.

n-Lorem, a nonprofit foundation, has announced the dates for its second annual Nano-rare Patient Colloquium, which will be held Oct. 30 & 31 in Cambridge, Mass. The Nano-rare Patient Colloquium will bring together a Community of Care for Nano-rare that consists of treating physicians, industry leaders, corporate, foundation and individual supporters, the n-Lorem team and most importantly, nano-rare patients and their families.

n-Lorem, a nonprofit Foundation, outlines the exceptional progress the Foundation has made toward its mission to discover, develop and provide treatment to nano-rare patients (1-30 patients worldwide), for free, for life. Already ten investigational new drug (IND) applications have been filed with the FDA and n-Lorem has dosed six nano-rare patients with plans to dose additional patients within the coming months.

n-Lorem, a nonprofit foundation, has released a limited edition holiday ornament that will support its mission to find and provide treatments for nano-rare patients (30 or fewer patients worldwide affected by a single gene mutation). Unveiled in advance of Giving Tuesday, the one-of-a-kind “I Heart Nano-rare” flat, shatterproof ornament will be sent to anyone who donates at least $25 to the organization. Donations to n-Lorem support the 100 patients already accepted for potential personalized treatments for free, for life and future patients. Already n-Lorem has filed nine investigational new drug (IND) applications with the FDA, begun treatment on five patients and plans to begin treatment on additional patients before the end of the year.

n-Lorem, a nonprofit foundation, announced Stanley T. Crooke, M.D., Ph.D., the founder and CEO of n-Lorem, has been honored as an Icon by the San Diego Business Journal with inclusion in the 2023 SD 500, a recognition that highlights the 500 most influential individuals in San Diego.

n-Lorem, a nonprofit foundation, will assemble patients, physicians, partners and supporters tomorrow, October 12, 2023, for its first-annual Nano-rare Patient Colloquium to provide valuable information to the nano-rare community, bring together those who support n-Lorem’s mission to finding treatments for nano-rare, give nano-rare patients and families a voice and provide a public platform to illustrate the high-quality process n-Lorem uses to advance from an accepted patient program to treating with an optimized ASO for that patient. Hosted at Biogen’s headquarters in Cambridge, Mass., the meeting includes more than 30 panelists and presenters and more than 250 in-person and virtual attendees, all of whom are dedicated to finding treatments for people with nano-rare illnesses (30 or fewer patients affected by a single gene mutation).

n-Lorem, a nonprofit foundation, today announce a collaboration with St. Jude Children’s Research Hospital to accelerate the development of optimized experimental antisense oligonucleotide (ASO) medicines for pediatric patients with extremely rare genetic neurological disorders.

n-Lorem, a nonprofit foundation, today announced that n-Lorem founder and CEO, Stanley Crooke, M.D., Ph.D., has been awarded the 2022 Steven C. Beering Award. This award recognizes Dr. Crooke’s seminal work in pioneering a new drug discovery platform that harnesses the power of antisense oligonucleotides (ASOs) to target genetic disorders and the field-changing implications that Dr. Crooke’s outstanding work has had, and will continue to have, in biomedical sciences.

n-Lorem, a nonprofit foundation, outlines the steps the Foundation has taken to support the discovery, development and treatment of nano-rare patients (1-30 patients worldwide), for free, for life. By industrializing the process, forming oversight committees, and performing every step in the process with the highest quality, n-Lorem is committed to ensuring that each nano-rare patient is exposed to only prudent risk, has the best, most optimized experimental antisense oligonucleotides (ASO), and is managed professionally throughout the entire process.

n-Lorem, a nonprofit foundation, today announced the publication of an article in Nature Biotechnology (Crooke, S.T., et al. Nature Biotechnology advanced online publication, July 2023). In this publication, Dr. Crooke and colleagues describe the diagnostic odyssey for Patient 001, n-Lorem’s first nano-rare patient accepted into the program, and provide a call to action for health care reforms to provide an integrated approach for delivery of care for these patients. n-Lorem is a nonprofit founded to discover, develop and provide personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

n-Lorem, a nonprofit foundation, celebrates a year of connecting with the nano-rare community through its patient advocacy initiative referred to as the ‘Patient Empowerment Program.’ A substantial component of this initiative is the podcast series hosted by Stan Crooke that was launched in May 2022. Through this podcast, n-Lorem is fostering a community for nano-rare patients by supporting and empowering them as they navigate understanding their disease. n-Lorem is a non-profit foundation discovering, developing and providing 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 it has been selected as a 2023 Edison gold award winner in the Treatment Optimization category for Health, Medical, and Biotech. This award recognizes n-Lorem’s innovative, non-profit approach to discover, develop and provide individualized, experimental ASO medicines for nano-rare patients (1 to 30 patients with the same single gene mutation worldwide) for free for life.

n-Lorem, a nonprofit Foundation, celebrates three years of progress in discovering, developing and providing experimental antisense oligonucleotide (ASO) medicines to nano-rare patients (1 to 30 patients with the same single gene mutation worldwide) for free for life.

n-Lorem a nonprofit Foundation, discovering, developing and providing experimental antisense oligonucleotides (ASOs) medicines to nano-rare patients (1 to 30 patients with the same single gene mutation worldwide) for free for life, today announced the newest additions to its Board of Directors – Paul Compton and Neil Shneider, M.D. Ph.D.

n-Lorem Foundation, a nonprofit dedicated to providing experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life, today announced that is has launched a new website to better serve patients, physicians and donors seeking information about the organization and how n-Lorem is helping patients with rare genetic illnesses.

n-Lorem, a nonprofit foundation, announced today that four investigational new drug (IND) applications have been filed successfully with the FDA and n-Lorem has begun dosing on three nano-rare patients. n-Lorem anticipates dosing the fourth patient in early 2023. Each patient is receiving a personalized, experimental antisense oligonucleotide (ASO) medicine discovered and developed for that single patient. n-Lorem’s mission is to discover, develop and provide personalized ASOs for nano-rare patients (1 to 30 patients worldwide) for free, for life.

n-Lorem, a nonprofit foundation, today announced that n-Lorem founder and CEO, Stanley Crooke, M.D., Ph.D. will receive the 2022 Roy Vagelos Pro Bono Humanum Award for Global Health Equity of the Prix Galien. This award recognizes Dr. Crooke’s nearly 50 years of dedication to a novel, clinically endorsed concept of using synthetic oligonucleotides to control the expression of genes and proteins that impact disease. Dr. Crooke is also recognized for his leadership in being among the first in industry to make a commitment to open science a key element of the modern R&D enterprise and to embrace the principle that even patients with the rarest of afflictions deserve the best talent and expertise the R&D industry can offer.

n-Lorem, a nonprofit foundation, today announced that Solve FSHD has provided one million dollars in support of n-Lorem’s efforts to fully understand the complex molecular cause of facioscapulohumeral muscular dystrophy-2 (FSHD2). This research will be critical to identifying a strategy for treatment for FSHD2 patients. In addition, this funding supports the expansion of n-Lorem’s research and discovery efforts, significantly enhancing the capabilities of the foundation.

n-Lorem, a nonprofit foundation, today announced the publication of an invited perspective article authored by n-Lorem founder and CEO, Dr. Stanley Crooke, and titled “Establishing an Environment in Which Rigorous Scientific Inquiry is Practiced” in Nucleic Acid Research (Crooke, S.T., NAR, Advanced Online Publication, July 2022). In this retrospective publication, the longevity and success of Ionis Pharmaceuticals is used to illustrate the unique challenges and rewards of establishing and fostering a corporate environment in which rigorous scientific enquiry is normal practice.

n-Lorem, a nonprofit foundation, announced today that Ultragenyx has continued its partnership with n-Lorem and provided a second donation to support n-Lorem’s efforts to discover, develop and provide 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 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.

n-Lorem, a nonprofit foundation, launches its patient advocacy initiative referred to as the ‘Patient Empowerment Program,’ with the release of its new podcast series. Through this program, n-Lorem hopes to foster a community for nano-rare patients by supporting and empowering them as they navigate understanding their disease. n-Lorem is a non-profit foundation discovering, developing and providing personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

n-Lorem, a nonprofit foundation, announced today the formation of Silence ALS, an initiative that aligns Columbia University and n-Lorem in support of the characterization of nano-rare amyotrophic lateral sclerosis (ALS) patients and the discovery and development of personalized experimental antisense oligonucleotide (ASO) medicines for these patients for free, for life. The initiative creates an infrastructure that employs n-Lorem’s nonprofit model to discover and develop personalized investigational ASO medicines for nano-rare ALS patients. Additionally, the initiative utilizes Columbia University’s resources to conduct a comprehensive clinical and scientific evaluation of pre-symptomatic ALS patients with the ultimate goal of identifying and treating nano-rare ALS patients prior to symptom onset.

n-Lorem, a nonprofit foundation, discovering and providing experimental antisense oligonucleotides (ASOs) to nano-rare disease patients (1 to 30 patients worldwide) for free for life, today announced that Amy McCooe has joined its Board of Directors.

n-Lorem, a nonprofit foundation, is the recipient of a $300,000 grant from The Conrad Prebys Foundation that will support n-Lorem’s discovery and development efforts to provide personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

n-Lorem, a nonprofit foundation, has engaged La Jolla Labs, a RNA drug discovery company, to support n-Lorem’s discovery efforts to provide personalized experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

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.

Argonaut Manufacturing Services a leading contract manufacturing organization, and n-Lorem, a nonprofit foundation, today announced a new partnership that supports and reduces the cost of manufacturing of n-Lorem’s experimental antisense oligonucleotide (ASO) medicines developed for 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 Alexion, AstraZeneca’s Rare Disease group, has made a donation of $1 million.

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 a new partnership with Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY).

n-Lorem, a nonprofit foundation, today announced the publication of a review article titled “Meeting the Needs of Patients with Ultra-rare Diseases” in Trends in Molecular Medicine (Crooke, S.T., Trends in Molecular Medicine, Advanced Online Publication, January 2022). In this publication, Dr. Crooke outlines the unique challenges of developing potentially life-saving treatments for a small number of ultra-rare patients, who because of their unique genetic mutation, are only one of a few patients worldwide. To distinguish this extremely small patient population, n-Lorem refers to these patients as nano-rare patients (1 to 30 patients worldwide). n-Lorem is the only non-profit foundation employing a charitable model that can meet the challenges of some of these nano-rare patients. n-Lorem uses a robust and proven ASO technology to discover and provide experimental antisense oligonucleotide (ASO) medicines for nano-rare patients for free, for life.

n-Lorem, a nonprofit foundation, today noted important new guidance recently issued by the U.S. Food and Drug Administration (FDA) regarding both the manufacturing processes and stability testing required for antisense oligonucleotides (ASOs) to be administered to nano-rare patients and the clinical administration of ASO therapeutics for nano-rare patients. The guidance titled “IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations” discusses the clinical development requirements for developing ASO therapeutics for patients with unique gene variants, making them a nano-rare patient population. 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.

n-Lorem, a nonprofit Foundation, discovering and providing experimental antisense oligonucleotides (ASOs) to nano-rare disease patients (1 to 30 patients worldwide) for free for life, today announced the newest additions to its Board of Directors – Jason Wood and Gene Yeo, Ph.D. and M.B.A.

n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicine to patients with ultra-rare diseases, today announced the appointment of Sarah Glass, Ph.D., as its first-ever Chief Development Officer.

n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients with ultra-rare diseases, today announced a new partnership with Nitto Avecia, the world’s largest oligonucleotide therapeutics manufacturer.

The editors of Nature Biotechnology recently published an editorial entitled “Ultra-precision medicine” in the June 2021 issue of Nature Biotechnologyi, highlighting new initiatives in n-of-1 drug development, as well as the rapid progress that n-Lorem Foundation, a nonprofit organization providing the ultra-rare disease community with free, lifetime supplies of individualized RNA-targeted medicines, has made since opening its doors in 2020.

n-Lorem Foundation, a nonprofit organization providing the ultra-rare disease patients with free, lifetime supplies of individualized RNA-targeted medicines, today announced the appointment of Claire Grezemkovsky as its first-ever Director of Philanthropy.

n-Lorem Foundation, a nonprofit organization providing the ultra-rare disease patients with free, lifetime supplies of individualized RNA-targeted medicines, today announced the newest addition to its Board of Directors – Frank Sasinowski, J.D., M.P.H., M.S.

n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients with ultra-rare diseases, today applauded important new guidance issued by the U.S. Food and Drug Administration (FDA) regarding the preclinical studies required for antisense oligonucleotide (ASO) treatment of patients with ultra-rare diseases. The constructive guidance is a positive and critical next step in meeting the needs of patients with ultra-rare mutations, and confirms the FDA’s stance that ASO technology is capable of helping “n-of 1” patients and is sufficiently advanced and understood to be subject to limited and expedited preclinical evaluation.

n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, today announced a new partnership with ChemGenes Corporation, a biotechnology company providing unparalleled expertise in the field of DNA/RNA synthesis for more than 40 years.