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n-Lorem Foundation receives additional support for providing "n of 1" experimental antisense medicines to treat patients with ultra-rare diseases
CARLSBAD, Calif., March 12, 2020 /PRNewswire/ -- The n-Lorem Foundation announced today it has received an initial pledge of $500,000 from Mark Lampert in support of the n-Lorem Foundation's efforts to provide advanced, experimental RNA-targeted medicines free of charge for life to patients living with ultra-rare diseases. Mr. Lampert is the founder and General Partner of BVF Partners L.P. ("BVF"), a San Francisco-based, private biotechnology investment firm established in 1993 that manages the Biotechnology Value Fund, L.P. and related entities.
"I'm proud to support the n-Lorem Foundation and their efforts to combine advanced genomic diagnostics with antisense technology to charitably meet the therapeutic needs of patients who suffer from extremely rare genetic diseases," said Mr. Lampert.
"Although patients with extremely rare genetic diseases, such as those with only 1-10 patients worldwide, present with tremendous challenges to traditional treatment approaches, antisense oligonucleotides (ASOs), provide an opportunity to efficiently create genetic medicines that are designed to correct certain specific genetic defects," said Dr. Stanley T. Crooke, chairman and CEO of n-Lorem Foundation. "I am deeply appreciative of this contribution from Mark, which adds to those from Ionis, Biogen and many others. I believe that with this support and that of the many others who have worked with me in this important effort coupled with the power of modern genomics and the antisense platform created by Ionis, we can responsibly meet the needs of some of these patients."
The n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably 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. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO and current executive chairman of the board of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.
To learn more about the n-Lorem Foundation, visit nlorem.org. If you are interested in supporting the efforts of the n-Lorem Foundation, please contact Tracy Johnson at 760-552-7113 or Tracy.Johnson@nlorem.org.
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SOURCE n-Lorem Foundation