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 investigator-initiated 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 investigator-initiated 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 an investigator-initiated 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?

View Guidance for submission

Does my patient
qualify for treatment?

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

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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