DATE

September 16, 2020

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Awards & Grants

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2020 – 2021 OTS Lifetime Achievement Award Winner Ryszard Kole, PhD

Ryszard Kole, PhD

Ercole Consulting

Dr. Kole received his Ph.D. degree from the Institute of Biochemistry and Biophysics, Polish Academy of Sciences, in Warsaw where he studied RNA the role of ribonucleases in cell function. He continued this line of work at Yale, in the Departments of Biology and Human Genetics, and during his 25-year tenure as Professor of Pharmacology at the UNC Chapel Hill. Dr. Kole’s activities span the gamut from biochemical assays, cell culture and animal studies to clinical trials as well as to methods of oligonucleotide delivery and improvements in oligonucleotide chemistry. Dr. Kole’s primary discovery was that pre-mRNA splicing provided a novel target for sequence specific therapies of severe disorders such as thalassemia, cystic fibrosis, DMD as well as cancer, inflammatory and metabolic disorders. It also represented a novel technology of oligonucleotide induced modulation of pre-mRNA splicing and exon skipping. [Dominski & Kole (1993) PMID: 8378346). His approach has huge therapeutic potential since over 90% of human genes produce alternatively spliced mRNA and about 50% of genetic disorders are caused by errors in pre-mRNA splicing. To commercially exploit this fertile field Dr. Kole founded Ercole Biotech, a company, which together with his group at UNC provided proof of concept in models of several rare diseases. In 2008 Ercole was acquired by Sarepta Therapeutics (previously AVI Biopharma), a Cambridge, MA company. At AVI and Sarepta Dr. Kole served as SVP Discovery and Distinguished Scientist. He is now a consultant to Sarepta. Sarepta’s breakthrough drug for DMD, (Exondys 51), approved by FDA 09/1/2016, is a direct result of Dr. Kole’s pioneering discoveries in oligonucleotide-induced modulation of pre-mRNA splicing. Dr. Kole’s approach was also explored by other researchers and companies some less (Glaxo/Prosensa) some more successfully (Spinraza, approved by FDA, 23/12/2016).