In this episode of "Rare Insights," host Taren Grom speaks with Dr. Joanne Donovah, Chief Medical Officer at Edgewise Therapeutics, about groundbreaking advancements for severe, rare muscle disorders. Dr. Donovan shares why Edgewise’s approach to targeting the basic unit of muscle contraction and being able to preserve and protect muscle function can have a positive impact on both Becker and Duchene patients. This episode also explores why leaning into understanding the patient voice can...

In this episode of "Rare Insights," host Taren Grom sits down with Dr. Linda Marbán, Chief Executive Officer at Capricor Therapeutics, to discuss groundbreaking advancements in the treatment of Duchenne muscular dystrophy and other rare diseases through innovative cell and exosome-based therapies. Dr. Marbán shares her journey in the biotechnology field, the unique approach of Capricor’s CAP-1002, and her vision for the future of rare disease therapeutics.   Discussion Topics: Advancing...

In this episode of "Rare Insights," host Taren Grom speaks with Dr. Jay Barth, Chief Medical Officer at Ascidian Therapeutics, about groundbreaking advancements in RNA editing technology and its potential in the rare disease space. Dr. Barth discusses Ascidian’s innovative approach, inspired by “sea squirts,” and shares insights on the development of ACDN-01 for Stargardt disease, an inherited form of macular degeneration. This episode also explores the challenges and opportunities in rare...

In this episode of "Rare Insights," host Taren Grom sits down with Dr. Kinnari Patel, President, Head of R&D and Chief Operating Officer for Rocket Pharma, to discuss the company’s burgeoning pipeline of rare disease assets and why applying data across its portfolio of potential treatments is just one key to the company’s success.   Dr. Kinnari Patel is President, Head of R&D, and Chief Operating Officer for Rocket Pharma, overseeing the global R&D and Technical Operations...

“Rare Insights” is a podcast series interviewing industry and organizational leaders about the development of  future therapies for rare diseases.   On “Rare Insights” we bridge the gap between those living with rare diseases and the biopharmaceutical industry. Know Rare amplifies the voices of individuals with rare conditions, providing invaluable perspectives to accelerate therapeutic solutions.    Join us as we dive deep into the complexities of rare diseases, exploring real-world insights...