A Unique Challenge

Congratulations on reaching n-Lorem. We are glad that you are considering joining our growing network of research physicians.

Meeting the needs of our patients

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.

The Patient’s Journey

Developing a personalized
ASO treatment plan

Each patient program will have a unique timeline. For some programs, drug discovery is relatively straight forward and an optimal ASO can be identified relatively quickly. For others, the discovery process can be longer and unfortunately, in some cases, we may not be able to identify an optimal ASO to move forward in development.

Learn more about the process.

Being Prepared

We are excited that you are considering partnering with n-Lorem. Before you begin the submission process, please see below for information on readiness, documentation for submission and expectations after submission. Because this is truly a partnership between n-Lorem, you and your institution, we will be working closely together to support the discovery, development and providing of a personalized experimental ASO for your patient.
Physician Readiness Requirements
  • Appropriate board-certified credentials in the therapeutic area that is relevant
    • Pediatric and adult neurology
    • Ophthalmology
    • Pulmonology
    • Hepatology
    • Nephrology
    • In born errors in metabolism
  • Experience in conducting clinical studies under INDs including Research INDs
  • Willingness and commitment to provide informed consent and care and evaluation consistent with best medical practices
  • Willingness and commitment to provide requested data to enable objective assessment of patient history
  • Willingness to work with a non-profit, n-Lorem, with no compensation
See below for information that is necessary to evaluate your patient. To begin, a detailed understanding of the genetics of the patient is critical to determining whether ASO technology is appropriate for a patient. As a general rule, full genome sequencing is most informative and needed. n-Lorem requires substantial information concerning the patient in order to make optimal decisions concerning the potential to treat a patient with an experimental ASO, including:
  • Diagnosis and the official name and symbol of the affected gene as defined by Gen Bank Accession Number and Gene ID.
  • Characterization of the mutation, including why this genetic change is unique compared to more common genetic changes associated with the diagnosis
  • Functionalization of the gene
  • Define the nature of the genetic change (gain of function, loss of function, splicing defect)
  • Define other genes that are altered
  • Clinical pathophenotype
  • Organs affected
  • Primary manifestations of the mutation to be treated
  • Other disease manifestations
  • Goals of the treatment (e.g. what symptoms do you expect to improve)
  • Added: Communicate progress of your patient’s program to the patient when appropriate

If accepted, collaboration with n-Lorem continues.

Given n-Lorem’s commitment the evaluation of the performance of each experimental ASO, it is vital to use the pre-treatment period to assure effective evaluation of the ASO.

Specifically, the research physician must:
  • In consultation with the patient or parent and n-Lorem, define a primary diseases manifestation and secondary manifestations of the disease to be treated and evaluated. These will be translated into the primary and secondary treatment goals outlined in the protocol.
  • Define specific measures of performance for each disease manifestation to be employed.
  • Develop a full treatment and evaluation plan.
  • Collect and document the baseline data of the disease in the months prior to treatment. The collected baseline data of the patient will then be summarized and provide to n-Lorem in a data transfer in a format mutually agreed upon.
  • Working closely with n-Lorem in the preparation and submission of the Research IND.
  • Engagement with the research physician’s institution regarding the conduct of a Research IND and related funding for the research protocol.
  • Meeting the requirements of the investigator’s institution prior to treatment. Each institution varies in the process required to engage in human experimentation and the information necessary to present to the institution’s IRB.
Each institution (tertiary care center) is a partner in the process and plays a critical role in ensuring that the patient receives the experimental ASO medicine in an efficient and timely manner once regulatory permission and IRB approval is granted and the experimental ASO medicine is ready to be administered.
Institutional readiness includes:
  • Commitment and experience in conducting clinical studies under a Research IND.
  • Commitment to providing the best medical care using an experimental medicine over a prolonged period of time.
  • Administrative systems to assure effective oversight including an experienced institutional review board (IRB) and experience in reporting adverse events and severe adverse events to the responsible institutions and regulatory agencies
  • Willingness to work seamlessly with a non-profit (n-Lorem) in assessing performance of experimental ASO medicines consistent with best medical practices
  • Willingness to perform care and investigation designed to be associated with minimal increases in cost to the institution without compensation

Are you ready to submit?

On the application for treatment page, you can request a new user account or login to start,
edit or submit an application

Does my patient
qualify for treatment?

Before submitting an application, you should review our qualifications to see if your patient meets the criteria.

Physician FAQs

Common questions from physicians answered

Experimental ASO treatments discovered and developed by n-Lorem are administered to n-Lorem patients according to a patient-specific clinical protocol under a Research IND held by n-Lorem.

  • These are not traditional clinical trials, and each IND and clinical protocol is customized to the individual patient.
  • n-Lorem does not provide funding or a clinical trial budget to physicians or institutions to cover the costs of treatment administration, physician time, pharmacy costs, and outcome assessments.

n-Lorem drafts >90% of the content of and submits all Research INDs, with the principal investigator being defined on the application. This reduces the regulatory burden on each site and streamlines submissions and FDA interactions for all n-Lorem patients.

  • Each physician will be expected to provide clinical content for the IND related to the patient and to sign off on the clinical protocol.
  • n-Lorem will manage all communication with the FDA for the lifetime of the IND including adverse event reporting and annual updates.

Each n-Lorem program is a collaboration between the physician who is requesting a treatment for their patient and n-Lorem. There are many important commitments for each physician who submits an application to n-Lorem and is interested in treating their patient with an n-Lorem therapy:

      1. Provide a cell line from your patient to n-Lorem for use during our drug discovery process. This may be fibroblasts or iPSCs depending on the gene target and anticipated expression levels in different tissue types. Physicians are responsible for paying for any costs with generating the cells and shipping them to n-Lorem.  No work will commence in our laboratory until the cells have been received.
      2. See your patient routinely in the clinic and provide updates from your visits (including clinic notes) to n-Lorem.
      3. Regularly meet with the n-Lorem team to provide updates on your patient’s clinical status and receive updates on the program’s progress to share with your patient.
      4. Communicate discovery and development updates directly to your patient and manage their timeline expectations using program specific information provided to you by n-Lorem. n-Lorem remains blinded to patient’s identities and does not provide updates to patients.
      5. Work with n-Lorem to establish individualized treatment goals for your patient and present your proposal to an outside committee for endorsement.
      6. Collect robust data using agreed upon outcome assessments both before and during treatment and share this data with n-Lorem.
      7. Contribute clinical content to the IND, obtain IRB approval for the study, and consent your patient.
      8. Work with your institution to secure adequate support and funding to be able to treat your patient.
      9. Administer treatment to your patient via the route of administration specific to your patient’s disease indication (ie. Intravitreal, intrathecal, or subcutaneous).
      10. Manage adverse events in collaboration with n-Lorem.
  • You will not be provided any financial compensation from n-Lorem for your involvement in treating your patient with an n-Lorem ASO.

Your institution will be expected to enter into a Treatment Agreement with n-Lorem. Drug cannot be shipped to your site until this agreement is executed.

  • There is no study budget provided as part of the Treatment Agreement because n-Lorem studies are not traditional clinical trials.

Your institution will be responsible for funding all costs associated with administering the treatment to your patient and performing clinical outcome assessments.

  • n-Lorem will provide the drug supply, for free, for the life of the patient as long as you as the treating physician deem a favorable benefit:risk ratio.

Many physicians / institutions cover these costs by:

  • Billing to insurance
    • Insurance coverage varies widely based on the provider and state of coverage.
  • Using philanthropic or research funds
  • Submitting grants
    • If you have a grant in mind to submit for, n-Lorem is happy to help provide necessary content to you and / or draft a letter of support.

There is no protocol that applies to all n-Lorem patients. Each clinical protocol is customized to the unique phenotype of each patient and all outcome assessments will be tailored to your patient in collaboration with n-Lorem.  However, we can provide an example schedule of assessments based on the anticipated route of administration to provide a high-level overview of the type of content our protocols typically include.

Applications are submitted through the n-Lorem portal “Matrix”.

  • Account requests are reviewed and granted once n-Lorem has confirmed each submitter’s understanding of their roles and responsibilities and the institutional infrastructure available to support our unique n of 1 study environment.

At this time n-Lorem can only treat patients who reside in the US. Applications for international patients will not be accepted although this may change in the future.

We encourage you to check back frequently to the n-Lorem website for updates on our efforts to expand beyond the United States.

Applications are reviewed in the order received. The completeness of each application upon receipt and timeliness to answer any follow-up questions or requests for additional documentation influence our review timelines.  In general, a decision on each application is made within 1-3 months of application receipt.

  • n-Lorem will initiate a unique ASO discovery and development program for your patient once all necessary data and materials have been received. The process to execute the necessary legal agreements and obtain the required data and cell material for each program can take between 3-12 months depending on the institution.
  • Once all materials are received, our rigorous discovery and development process takes a minimum of 18 months. However, this timeline can vary substantially based on the gene target of interest and complexity of the program. n-Lorem will provide routine updates to you along the way which can be shared directly with your patient.
  • It is important to note that for some programs we will not be successful in identifying a therapy for the patient.
  • In some cases, an ASO may be available for your patient already. In these situations, we will communicate directly with you regarding the regulatory strategy for treatment.
  • Depending on the planned ASO strategy, your patient’s program may require long read whole genome sequencing (WGS) data. n-Lorem has a vendor who can consent your patient and provide the sequencing at no cost to you or the family if your institution cannot generate this data in-house. Requirements for sequencing will be communicated directly to you and you will be responsible for connecting your patient with our sequencing provider, as needed. Once sequencing data is available, the n-Lorem sequencing provider will send the data directly to n-Lorem for analysis.
  • Most programs in our laboratory require patient cells for our experiments, and we cannot move a program forward in our laboratory until all required cells have been received. The cell type needed will be dependent on the anticipated expression levels of the gene target of interest. Requirements for the cell type will be communicated directly to you, and you will be responsible for coordinating and funding sample collection and generation of any necessary cell lines. Once a Material Transfer Agreement (MTA) is executed, we will arrange for shipment of the patient cells to n-Lorem.
  • Before a program can move forward in our laboratory, a technical review is required to determine if ASO design will be feasible. Once sequencing is completed, n-Lorem scientists will evaluate the patient’s genetic information to determine if ASO design is possible. n-Lorem will update you when this review has been completed.
  • Once the technical assessment for your patient’s program is complete, n-Lorem will work with you to define the individualized treatment goals and relevant outcome assessments that will be used to measure those goals. These goals and assessments will drive the framework of your patient’s individualized clinical protocol.
  • It is expected that you will collect at least 1 year of data pre-treatment using the agreed upon outcome assessments.
  • Each treatment protocol will also require at least 1 year of on study data collection.
  • Data on the clinical outcome assessments according to the protocol is required to be collected as long as the patient remains on treatment with the n-Lorem ASO unless a different strategy is discussed and agreed with n-Lorem.
  • All data will be shared with n-Lorem via a REDCap database that will be built specifically for your patient by n-Lorem.

n-Lorem ASOs for CNS indications are administered intrathecally.  Dosing typically occurs at Day 1, Month 1, Month 3 and quarterly thereafter. At the first dose and each dose escalation (4-5 visits in the first year of treatment) an overnight hospital stay is required for observation.  Once we have reached a maintenance dose on a quarterly schedule, the observation period becomes 6 hours vs. overnight.  The dosing regimen will be precisely defined for each patient’s ASO.

  • As a non-profit organization, n-Lorem does not provide clinical trial funding to sites to cover the costs of drug administration and hospital stays.

The route of administration for ophthalmologic indications is intravitreal with dosing typically occurring every three months, however it will be precisely defined for each patient’s ASO.

  • As a non-profit organization, n-Lorem does not provide clinical trial funding to sites to cover the costs of drug administration.

The route of administration for systemically delivered ASOs is subcutaneous with dosing typically occurring monthly, however it will be precisely defined for each patient’s ASO.

  • As a non-profit organization, n-Lorem does not provide clinical trial funding to sites to cover the costs of drug administration.

More questions?
Please reach out to us!

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

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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