The n-Lorem Foundation will collaborate with St. Jude Children’s Research Hospital to speed up development of antisense oligonucleotide (ASO) medicines for children with very rare genetic conditions.
The two organizations will begin the partnership by developing two ASOs together for the treatment of very rare genetic neurological disorders over several years with a view to speeding up n-Lorem’s development of ASOs and expanding St. Jude’s recently established genomic medicine program.
“This collaboration is truly transformational and has the potential to expand the number of patients with extremely rare diseases that could benefit from an optimized personalized ASO medicine,” said Stanley Crooke, founder, chairman and CEO, n-Lorem Foundation, in a press statement.
“By creating a model that is set apart by the rigorous processes n-Lorem has in place and leveraging the teams that created ASO technology to teach others, we are able to work with other high-quality institutions, like St. Jude, so that together, we can accelerate the development of therapies for more patients.”
Crooke was founder and executive chairman of Ionis Pharmaceuticals, a company focusing on antisense RNA-targeted therapeutics, for over 30 years. He set up the non-profit n-Lorem Foundation in 2020 with his wife Rosanne Crooke and, since retiring in 2021, has led the organization.
The goal of n-Lorem is to help patients with what they call ‘nano-rare’ genetic conditions that impact between one and 30 patients around the world. Patients who qualify for treatment with ASOs will be provided with free, individualized and life-long treatment by the non-profit, which now has funding from more than 75 sources including initial funders the Crookes, Ionis Pharmaceuticals and Biogen.
So far, n-Lorem has received over 225 applications for treatment from patients with nano-rare diseases. Of these, around 100 patients are having individualized treatments developed for them by the organization.
As of May this year, three patients had received n-Lorem treatments and another five were scheduled to be treated later in the year. The non-profit has a five-year goal to have 200 patients under treatment.
By collaborating with St. Jude Children’s Hospital, which also has a focus on treating children with extremely rare disorders, n-Lorem hopes to speed up its ASO pipeline. It will train scientists at St. Jude to discover and develop optimized ASOs for treatment of rare disease and, in return, the hospital researchers will provide these services to the non-profit in the future. This collaboration should reduce the cost of developing these medicines to n-Lorem and allow the non-profit to treat more nano-rare patients.
