Session Summary
Keynote presentation by Stan Crooke, Executive Chairman of Ionis Pharmaceuticals
Summarized by biotech researcher and writer Catarina Carrao
N-Lorem is a foundation created by Dr. Stanley Crooke, Executive Chairman at Ionis Pharmaceuticals, a year ago to create individualized treatments for patients with ultra-rare diseases using the technology developed at Ionis Pharmaceuticals. N-Lorem’s mission is to apply efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotides (ASO) medicines to treat patients with ultra-rare diseases (<30 patients).
The n-of-1 patient is a large and growing population with desperate needs. Though each patient has a unique mutation, millions of n-of-1 patients have been identified; and, the more genomic sequencing is completed, the more n-of-1 patients will be identified. Within the larger populations of genetically caused diseases, there is a common mutation and patients with unique mutations. Though great progress is being made in identifying these patients and finding the genetic causes of their diseases, drugs that work in patients with the common mutations often do not work for n-of-1 patients. N-Lorem is currently the only solution to treat many of these patients.
So far, Ionis and n-Lorem have helped design and provide experimental ASOs for two patients: one with Batten’s disease and ataxia-telangiectasia (AT), the other one with a FUS mutation in an ALS background. These have provided an excellent opportunity to validate the n-Lorem concept. The first step toward potential treatment is to achieve a diagnosis and full genetic analysis. Then the patient needs to work with a physician that connects directly with n-Lorem, who will make an informed decision about whether a patient is appropriate to receive an experimental ASO treatment. It is critical that the patient be cared for by a qualified investigator and a qualified institution. Upon completed review, an ‘Access to Treatment Committee’ makes recommendations to the n-Lorem’s executive team with regard to the appropriateness to treat.
Proposals are approved and prioritized based on criteria such as: the severity of the disease, feasibility of developing an ASO treatment for the genetic cause of the disease, degree of potential benefit vs. potential risks, practicality of treatment, availability of physician and institution to treat patient and conduct necessary studies, and other complexities of the condition. The FDA response to the n-Lorem concept has been very supportive, and initial guidance is already in place since January 2021. As such, upon regulatory permission, an investigator-led clinical trial is initiated and then a patient may receive their custom experimental ASO treatment at no cost.
Gratuitous individualized therapy is feasible for Ionis Technology because of its intrinsic technological efficiency and versatility. Our understanding of modern ASOs mechanisms and potency in selected organs, with different routes of application, together with Ionis automation and integrated safety databases, allows a leap for the treatment of unique patients. This is a great opportunity to do something good for families in need and also for the industry.