Welcome & Opening Remarks

Keynote Presentation:

n-Lorem, a dream of hope and treatment for nano-rare patients realized

n-Lorem exists because we believe that all patients deserve the opportunity to be treated, irrespective of the rarity of the disease or the economic status of the patient or family. In this presentation, Stan provides an overview of our progress, what we are learning from all of our patients and what it takes to create a truly life-changing organization committed to offering free ASOs to patients for life.

Supporter Panel:

Helping n-Lorem do more

At n-Lorem, we focus on patients who have diseases caused by a single gene mutation that is found in no more than 30 patients in the world. We choose this patient population because today there is no path to commercial approval or commercial model that can address the needs of such rare patients and because we hope that we can raise sufficient donations to treat as many nano-rare patients as we are able to. Already, the need has exceeded our expectations, and we need more to do more. In this panel, some of our supporters discuss how as a community we can do more for nano-rare patients.

Foundation Panel:

Expanding the number of patients treated with high-quality ASOs

A core belief at n-Lorem is that every patient matters. The first dictum of therapeutics is to treat the patients who can be treated today while investing long term to identify better solutions for more patients. In this panel, opportunities are discussed to expand the number of patients we can treat by working closely with appropriate institutions, patient advocacy groups and regulators outside of the U.S. We are committed to enhancing health care and changing the world one patient, one family at a time.

Patient Journeys:

The perilous journey to diagnosis and treatment for nano-rare patients

n-Lorem exists to serve the nano-rare patient community. The opportunity for ASO treatment provides hope to the hopeless and eventually, we hope, treatments for many. Hopelessness and despair that come at the end of a long and perilous journey to diagnosis are devastating. In this special panel, some of our nano-rare patients share their diagnostic odysseys and what n-Lorem means for them.

Presentation:

Driving innovation in rare disease

Partner Panel:

What it means to be a partner with n-Lorem for nano-rare

The challenges presented by nano-rare patients are far too complex for n-Lorem to solve even a fraction of the issues without support. Thus, our goal from inception has been to build as broad and effective a network of contributing stakeholders. In this panel, our partners in our community of care discuss what it means to be a partner with n-Lorem. Including how partnering with n-Lorem provides their employees with a feeling of purpose.

Hero of n-Lorem Awards

The ‘Hero of n-Lorem’ Award is a recognition given to an individual volunteer or organization that has made substantial contributions to n-Lorem and our nano-rare patients. This honor recognizes our ‘unsung heroes’, those who have gone the extra mile, taken time away from their schedules and families and leveraged their expertise and contacts to support n-Lorem’s mission to provide hope and potential help to nano-rare patients.

Presentation:

ASO experience and scientific excellence driving the n-Lorem discovery and development platform

Every new patient represents a novel ASO discovery and development program. The essence of science is uncertainty and the process of discovering a novel medicine is complex and arduous, even for ASOs. At n-Lorem, we are industrializing the treatment of nano-rare patients because we believe that each patient deserves the very best ASO that we can discover and develop. In this presentation, we provide data driven examples of hard choices that only expertise, a deep understanding of ASOs and decades of experience developing ASOs into a powerful drug discovery platform, can inform. Our patients deserve nothing short of the best that we can do.

Presentation:

n-Lorem clinical infrastructure assures professional management of our patients

As our mission is entirely focused on helping nano-rare patients, the single most important area of progress will be to learn maximally from each patient. In this presentation, we outline the infrastructure we have in place to ensure every step in the process be of the highest quality possible if we are to maximize the opportunity for benefit and minimize the risk of adverse events in our patients. We discuss how we professionally manage exposure of each patient to an experimental medicine, accumulate, analyze and share data and the exciting data what we are seeing from our patients today.

Presentation:

An integrated operational model for the nano-rare

n-Lorem integrates a powerful, patient-driven mission, with a robust, versatile technology that enables the discovery and development of optimized personalized ASO medicines for nano-rare patients. n-Lorem has brought this mission to fruition through the establishment of an integrated operational model. As a non-profit, every new efficiency in the processes or new way of thinking by the n-Lorem team leads directly to our ability to help more nano-rare patients. In this presentation, Sarah Glass discusses how all these pieces integrate to create a scalable and robust model dedicated to helping the patients it can help today, expanding to help more patients tomorrow and advancing the science to broaden its opportunities throughout the broader ASO population.

Physician Panel:

A physician’s perspective on n-Lorem and nano-rare

It is vitally important for n-Lorem to work with scientists and physicians to ensure that patients are exposed to only optimized personalized ASO medicines. We are lucky to have world-class renowned physicians supporting our patients and helping us at every stage of the program’s development. In this assembly of medical professionals, our treating physicians discuss their experience with n-Lorem and how they support their patients throughout the process.

Presentation:

Empowering nano-rare patients

One truly is “the loneliest number” and most nano-rare patients are exactly that: the single patient in the world with their unique mutation and disease. Whether a nano-rare patient is truly n-of-1 or a member of a tiny patient population, the patient is entirely isolated. In response, we are developing an array of pertinent support efforts that will seek to create a sense of community for nano-rare patients. In this presentation, Amy Williford discusses our Patient Empowerment Program and how we are creating a community of care.

Supporter Panel:

Learning from nano-rare patients

Advances in science are incremental and each additional bit of knowledge contributes to the evolution of understanding. No area of scientific inquiry is more incremental than advances in the treatment of diseases. What can be learned by addressing the needs of patients with the rarest of mutations that cause a more common rare genetic disease is immediately applicable to the entire group of patients with the disease. In this panel, we discuss how studying a patient or a few patients, even if they have the rarest of the mutations known to cause the disease, can expand our knowledge about the molecular causes of a disease and potential value of therapeutic interventions.

Presentation:

Healthcare reforms urgently required to meet the needs of nano-rare patients

In a developed economy, access to quality health care is a right, and at n-Lorem, we do our best for every single patient every single day. There are some obvious reforms needed that are outlined in this presentation. We hope that more will join us, as we strive to enhance health care for nano-rare patients and change the world one patient, one family at a time.

Closing Remarks