n-Lorem Foundation receives $500,000 Pledge for ASOs to Treat Ultra-Rare Diseases

The n-Lorem Foundation, the nonprofit founded by antisense pioneer and Ionis Pharmaceuticals Executive Chairman Stanley Crooke to provide experimental RNA-targeted therapies for patients with ultra-rare diseases free of charge, said it received a pledge of $500,000.

Paul and Andrea Compton, founders of CureDRPLA made the commitment. CureDRPLA is a foundation focused on finding a cure for dentatorubral-pallidoluysian atrophy, a rare neurodegenerative disorder. Paul Compton is president of Barclays Bank.

“As parents of a child who suffers from a rare neurological disease, we believe deeply in the mission of n-lorem and are very pleased to be able to make this contribution in support of its continued success,” the Comptons said in a statement announcing the donation.

The n-Lorem Foundation is applying antisense technology to provide experimental antisense oligonucleotide (ASO) medicines to treat patients with ultra-rare diseases (1 to 10 patients) that are the result of a single genetic defect unique to only one or very few individuals.

ASOs are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific.

Crooke, chairman and CEO of the n-Lorem Foundation, welcomed the donation and said that every contribution means that n-Lorem can do more for needy patients and their families.

“These patients present significant challenges that are often difficult for traditional treatment approaches to address,” he said. “In contrast, the antisense oligonucleotide platform created by Ionis provides an opportunity to efficiently create genetic medicines designed to correct specific genetic defects.”

Photo: Stanley Crooke, chairman and CEO of the n-Lorem Foundation

Author: Rare Daily Staff