#141 Stan Crooke on Ultra Rare Disease Drugs

Biotech titan Dr. Stan Crooke joins host Kira Dineen to celebrate rare disease month! Dr. Crooke was the Founder of IONIS Pharmaceuticals, with extensive experience in the pharmaceutical industry developing more than 20 marketed drugs. He has published nearly 500 scientific publications, edited more than 20 books, and has numerous patents.  Dr. Stan Crooke is now the Founder and CEO of n-Lorem Foundation, a new San Diego-based organization with an incredible mission of developing individualized RNA targeted medicines for patients with ultra-rare diseases, and providing those treatments for free, for life. These patients have extremely unique mutations and are often only one of 30 people in the entire world to have the disease. After only one year as a foundation, they’ve already made great progress for the ultra rare community, having received 50 applications from patients with ultra rare genetic mutations. Out of those 50, they have greenlighted treatment plans for nearly 20 patients - greatly exceeding application and acceptance rate expectations.  On This Episode We Discuss: Rare diseases vs ultra rare diseases Challenges treating patients with ultra rare diseases and genetic mutations Standard process and cost of drug development n-Lorem’s new approach to drug development for ultra rare diseases  Antisense therapies (ASOs) n-Lorem’s charitable and scalable model  n-Lorem’s relationship with IONIS Pharmaceuticals People eligible for n-Lorem’s treatments How to contact n-Lorem’s for potential treatment  Drugs currently in development at n-Lorem Insight on the development of SPINRAZA® for spinal muscular atrophy  Learn more about n-Lorem on their website.  Check out the UConn Podcast Symposium, our host Kira Dineen will be on the interdisciplinary panel taking place on February 22nd at 4PM EST. You can register to attend for free here. UConn students will be provided a Zoom link to engage in a live Q&A. The panel will also be streamed publicly via Facebook and YouTube. Stay tuned for the next new episode of DNA Today on March 5th, 2021! New episodes are released on the first and third Friday of the month. In the meantime, you can binge over 140 other episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “DNA Today”. Brand new in 2021, episodes are now also recorded with video which you can watch on our YouTube channel.   See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, DNApodcast.com. Questions/inquiries can be sent to info@DNApodcast.com.