Antisense pioneer
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
Author: Rare Daily Staff
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