Rare Daily Staff
and Ionis Pharmaceuticals Executive Chairman Stanley Crooke has launched a
nonprofit to provide experimental RNA-targeted therapies for patients with
ultra-rare diseases free of charge.
The organization, n-Lorem Foundation, is backed with funding from Crooke and his wife Rosanne, an Ionis researcher. Ionis and Biogen are providing additional funding as the first corporate donors, in addition to support from individual contributors.
Crooke, who is serving as the chairman and CEO of n-Lorem, drove the development of Ionis’ antisense platform. Antisense oligonucleotides (ASOs) are small pieces of chemically modified genetic material designed to bind to target RNAs for therapeutic use. They can be developed rapidly, inexpensively, and are highly specific.
He said the n-Lorem Foundation is designed to couple advanced genomic diagnostics with antisense technology to charitably meet the therapeutic needs of patients with ultra-rare conditions.
“No single charitable foundation or drug discovery technology can meet the needs of all the patients who suffer from ultra-rare diseases, but having led the creation of the antisense platform,” he said, “I believe that by bringing together the power of modern genomics and the antisense platform created by Ionis, we can responsibly meet the needs of some of these patients.”
Patients with a diagnosis and full genetic analysis can have their physician submit a proposal for treatment to n-Lorem. The organization’s Access to Treatment Committee will review proposals for treatment and make a recommendation to the foundation. The organization said it will approve and prioritize patients based on 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.
If approved, the foundation will work with Ionis to develop an ASO for the patient, perform studies to find the best ASO candidate, and work with clinical investigators and regulators to obtain necessary approvals to test and treat the patient.
Photo: Stanley Crooke, Chairman and CEO of n-Lorem Foundation