Imagine you are one of only 10 people in the world with a disease. People with ultra-rare conditions have little hope of attracting research funding to find treatments to work for them. The investments just don’t make sense to for-profit businesses.
Our guest, Dr. Stanley Crooke, MD, PHD is leading an effort to change this. After an illustrious career in pharma and founding the RNA Therapy company Ionis Pharmaceuticals, he is blazing a new trail. He has foundedn-Lorem, a non-profit organization that works with families affected by these ultrarare conditions. N-Lorem works to find, develop, and provide new RNA treatments for free, for life.
RNA therapy, which includes anti-sense oligonucleotides, has been a dream for researchers for decades. Dr. Crooke has been at the forefront of this work. Now that years of labor are starting to bear fruit, he is looking to a new chapter. A way to give back. The question has changed from, “Can We?” to, “How can we not?”