Providing hope for a better future, one nano-rare patient at a time | NRPC24

Описание: Innovative Treatments for Nano-Rare Diseases

Stanley T. Crooke, M.D., Ph.D., n-Lorem’s Founder, Chairman, and CEO, as he discusses how n-Lorem is at the forefront of providing innovative treatments for nano-rare patients. In this presentation during the 2024 Nano-rare Patient Colloquium on October 30 in Cambridge, MA, Stan explains how n-Lorem is delivering hope and making a tangible impact on the future of nano-rare disease care, one patient at a time.

The n-Lorem Foundation is a nonprofit focused on discovering, developing, and providing personalized ASO treatments for patients with nano-rare diseases (1 to 30 patients worldwide), for free, for life.

Dr. Crooke founded and was Chairman and Chief Executive Officer and Lead Scientist of Ionis Pharmaceuticals. During his tenure at Ionis, he led the scientific development of a new platform for drug discovery, antisense technology and the creation of one of the largest and more advanced development pipelines in the biotechnology industry, and commercialized several antisense drugs including, SPINRAZA® (nusinersen), TEGSEDI™ (inotersen) and others. Early in Dr. Crooke’s career, he led the creation of the first broad anticancer program in the industry at Bristol-Myers, bringing numerous anticancer drugs to the market in the first five years of his career. He then assumed responsibility for worldwide R&D (president) at SmithKline Beckman (now GSK). During his tenure at SKB, Dr. Crooke led the restructuring of R&D and the development of several drugs that were commercialized.

Dr. Crooke has also contemporaneously led a successful academic career becoming a full professor at Baylor College of Medicine and the University of Pennsylvania Medical School where he trained a number of Ph.D. students and won several teaching awards. Dr. Crooke has been an active scientist throughout his career as well.

Dr. Crooke has received a number of awards, most recently, the Indiana University School of Medicine Steven C. Beering Award, the Prix Galien Roy Vagelos Pro Bono Humanum Award, the American Chemical Society’s E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances, the Lifetime Achievement Award presented by the Oligonucleotide Therapeutics Society, the Scrip Lifetime Achievement Award and the 2019 Massry Prize.

Dr. Crooke received his M.D. and Ph.D. degrees and house staff training at Baylor College of Medicine, where he currently serves on the Board of Advisors. In 2021, Dr. Crooke has been named Distinguished Alumnus of both Baylor College of Medicine’s Graduate and Medical schools and named one of the 20 of the most influential biopharma R&D executives by Endpoints News. He has published nearly 600 scientific publications, edited more than 20 books, has numerous patents, and led the development of more than 23 drugs that have been commercialized.

During the Colloquium, we reflected on n-Lorem’s progress as the foundation continued to expand. We shared the latest developments, our evolving vision for the future, and the knowledge gained over the years in the fight against nano-rare diseases.

We highlighted the processes that allowed us to efficiently develop optimal personalized ASO medicines tailored to individual patients. Patient families shared their emotional journeys from diagnosis to treatment, while our partners discussed how supporting nano-rare initiatives enriched their company cultures and fostered collaboration in the rare disease space.

Additionally, supporters shed light on the growing challenges n-Lorem faced as more nano-rare patients reached out for help. Leading research physicians from across the country provided insights into the latest findings in neurological diseases, offering a deeper understanding of the current landscape of rare disease research.

https://www.nlorem.org/nano-rare-pati...