Welcome to n-Lorem

Congratulations on reaching n-Lorem. You are at the cutting edge of personalized medicines and have the opportunity to support nano-rare patients.

Making a lasting impact

At n-Lorem, we are blazing new ground in the treatment of nano-rare patients. We are the first organization that leverages a mutation-driven approach to target the unique genetic cause of disease. We bring knowledge, technological expertise, high quality systems, professional clinical management and a team of dedicated, compassionate professionals to provide only the best, most optimized medicine.

Opportunities for Support

Why your institution should collaborate with us in treating patients with nano-rare disease

Nano-rare patients are the rarest and most underserved patient population in the developed economy. The fortunate few who are diagnosed undergo prolonged, perilous and extraordinarily costly diagnostic journeys replete with misreferrals, misdiagnosis and mistreatment. We off the opportunity to provide treatment for free, for life, but we can only do that with your help.

Opportunities for Research

What we learn today can change the way we think about disease

Nano-rare patients also provide an extraordinary opportunity for research. Virtually nothing is known about nano-rare patients and yet they represent a great opportunity to evaluate the impact of single gene mutations on homeostasis and the evolution of symptomatology. This knowledge will be readily publishable and have a direct impact on overall understanding of health and disease. Further, the work provides the opportunity to examine the impact of genetic medicines on targets, pathophysiology and patient disease. In short, working with n-Lorem provides a great opportunity to serve patients in a fashion consistent with the mandate of tertiary care institutions.

Getting Involved

Discovering, developing and providing personalized experimental ASO medicines to nano-rare patients is a collaborative effort between n-Lorem, the research and/or treating physician and the institution where the patient will be treated. A collaborative engagement between us and each institution is a critical component of ensuring the most efficient path to treating your patient. We have established quality systems and processes for clinical and regulatory operations with associated support that will enable this effective collaboration.
Both treatment evaluation and master transfer agreements are key
components of the n-Lorem/Institution relationship.
  • Treatment Evaluation Agreement

    We are committed to discovering, developing, and providing the personalized ASO to your patient for free, for life- provided we and the physician deem a favorable benefit:risk ratio. Because these are n of 1 or n of few treatment evaluations, it is also our commitment to learn as much as possible from each patient. This Agreement is critical to ensuring terms surrounding data sharing, accountabilities and risk are agreed upon before treating a patient.

    Upon acceptance into the program, we will send a template for the Treatment Evaluation Agreement to the Investigator. The n-Lorem team and legal counsel will work with your Institution’s legal counsel to achieve a mutually agreeable document in a timely fashion. This Agreement can serve as a framework for future patients as well.

  • Material Transfer Agreement

    Depending on the ASO strategy, the n-Lorem ASO discovery team may require cells from your patient to support our in vitro screening studies. Upon acceptance into the program, the n-Lorem ASO discovery team will determine the necessity for your patient’s cells which may be fibroblasts or iPSCs. If cells are needed, we will execute a Material Transfer Agreement (MTA) between n-Lorem and your institution so that we may receive your patient’s cells. It is the physician’s responsibility to obtain any necessary IRB approvals for sample collection and to generate the required cell lines.

The Investigator’s ability to make the benefit:risk decision for a patient when on treatment is dependent entirely on having pre-treatment and during-treatment clinical outcome assessments and associated data taken in a timely and high quality manner. Upon acceptance into the program, n-Lorem and the Investigator will collaborate to define the treatment goals and establish the clinical outcome assessments required to evaluate the progress towards achieving the treatment goals. The treatment goals will be translated into the patient-specific protocol. As per the requirements outlined for research INDs, investigators must maintain adequate and accurate case histories, specifically that patient data must be properly captured. We have established a CFR Part 11 Compliant REDCap platform (n-Lorem RedCap) that will enable efficient data collection and remove the burden of establishing the REDCap from the institution. We also have a Clinical Data Scientist and Medical Monitor responsible for supporting the Investigator to collect this data.

Today we are only treating patients in the United States, but plan to expand to other countries. We are able to treat in the United States because of regulatory guidance issued by the FDA. Patients are treated with n-Lorem individualized ASOs under Research Investigational New Drug (IND)s. n-Lorem will be the IND-holder and will be responsible for submitting the IND to the United States FDA and will meet safety and annual reporting requirements.

The n-Lorem data platform will enable efficient extraction of key data annually to support the Investigator’s annual reporting requirements.

n-Lorem has a successful track record of all Research INDs having been approved. The content of the Research IND is primarily pre-clinical data regarding the experimental ASO and the n-Lorem R&D team will author all appropriate sections in accordance with the FDA requirements as defined in the 4 guidance documents.

Each Investigator is responsible for meeting all required regulatory reporting obligations and will submit adverse event reports to the FDA in accordance with applicable laws and regulations. The investigator will also inform n-Lorem of any adverse events in a timeline consistent with FDA notification requirements by providing a copy of the report submitted to the FDA. We will follow all safety and regulatory requirements per the FDA guidelines and local regulations. Additionally, we will share safety updates as an element of professional management of the exposure of patients to experimental agents.
Each Institution is required to obtain approval (and continuing review) for each Research IND by an Institutional Review Board (“IRB”), meet any other institutional requirements, comply with all terms of IRB approval and provide timely reports as requested by the IRB. The initiation of treatment at each Institution will not begin until the investigator receives a May Proceed Letter from the FDA and IRB approval is obtained. The investigator will keep the IRB fully informed of the progress of treatment. The n-Lorem Clinical Operations team will support the Investigator with meeting all Institution-specific IRB requirements for documentation in advance of the treatment as well as during treatment, as requested.

Nano-rare patients present a unique opportunity to learn about health and disease, and we are obligated to learn maximally from each patient and our aggregate experience and share our learnings with all interested parties as rapidly as possible. Our data platform will enable efficient extraction of key data annually to support the Investigator’s annual reporting requirements.  Our data platform will also enable the publishing of case reports and aggregate data.  See all relevant peer-reviewed publications, including safety database papers, which aggregate safety data on over three decades of experience with antisense technology.


n-Lorem is committed to transparency and data-sharing.

The clinical data collected during treatment will be collected in a  Title 21 CFR Part 11 and HIPAA compliant data platform in a high-quality and rigorous manner.  Pre-treatment and baseline data will be shared in a mutually agreed upon format (i.e. STDM or CVS file transfer).  As noted earlier, obtaining the baseline data along with the treatment data will enable the investigator to evaluate the clinical outcome assessments required to determine continuation of treatment.

Preclinical data from the ASO discovery and development will be shared in peer-reviewed publications when the totality of the data can be interpreted in a meaningful way.  Because of the commitment to quality for every patient’s ASO, the correlation between the ASO characteristics and R&D process and the clinical outcomes will be assessed across the portfolio of n-Lorem ASOs and published.

We are obligated to continuity of care for our patients. n-Lorem is committed to manufacturing sufficient drug supply for each patient for as long as the benefit:risk is deemed favorable by the Investigator and n-Lorem. If there are any changes in the patient’s location or the Investigator’s location or ability to administer the n-Lorem ASO, the Institution agrees to notify n-Lorem promptly and collaborate with n-Lorem to identify another institution and/or treating physician to provide continued care.
As a nonprofit institution that intends to provide experimental ASO medicines for free, for life to nano-rare patients, we have no intention of commercial development for our medicine. Because our resources are earmarked for discovery, development and manufacturing of the experimental ASO medicine, we cannot pay tertiary care institutions to cover the costs associated with providing data to allow assessment of the performance of the ASO medicine. Because of this, we ask only that the physician and institution perform studies that are consistent with best care practices. That said, if a physician or institution wishes to conduct additional research studies, we welcome those efforts as well.

More questions?
Please reach out to us!

For additional information — please see our Frequently Asked Questions or reach out to us at [email protected].

The Patient’s Journey

Developing a personalized ASO treatment plan

At n-Lorem, our mission is to treat the patients we can today. By working closely with our research physicians and their institutions, we discover, develop and provide personalized experimental ASO medicines to nano-rare patients for free, for life. This journey begins with a research physician submitting an application to treat for their patient to n-Lorem.

Learn more about ASO technology and its potential for treatment

We cannot do
this alone

Together we are changing the world—
one patient at a time

We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.

We need your support

Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.

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