#4 Conquering the Science of Muscle Disease with Edgewise Therapeutics

Rare Insights: Uncovering The Future Of Rare Disease Treatments

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 benefit drug development overall.

Joanne M. Donovan, M.D., Ph.D., has been Chief Medical Officer at Edgewise Therapeutics, a biotechnology company focused on rare muscle disorders, since April 2021. Dr. Donovan has been deeply involved in clinical development for Duchenne muscular dystrophy for the last decade in her role as Chief Medical Officer and Senior Vice President, Clinical Development at Catabasis Pharmaceuticals. From 1998 to 2011, Dr. Donovan served in positions of increasing responsibility, ultimately as vice president of clinical development, at Genzyme. Since 1989, she has been a staff physician at the VA Boston Healthcare System and is currently an Associate Clinical Professor of Medicine at Harvard Medical School. Dr. Donovan holds a Ph.D. in medical engineering and medical physics from the Massachusetts Institute of Technology, an M.D. from Harvard Medical School and an S.B. from the Massachusetts Institute of Technology. She completed residency training in internal medicine and a fellowship in gastroenterology at the Brigham and Women's Hospital.

Discussion Topics:

1. Introduction to Edgewise Therapeutics: Exploring the company’s foundational experience in small molecule engineering and expertise in muscle biology
2. Understanding Cellular Machinery: Preserving and protecting muscle function can translate to improved outcomes
3. Empowering the Patient Community: By listening to patients’ voices, there is an opportunity to enhance the foundation of drug development
4. Technology Advancement: Incorporating remote access tools can lessen the burden of disease for patients during clinical trials
5. Living the Legacy in Rare Disease: Dr. Donovan’s experience while at Genzyme has shaped her approach to understanding and overcoming the barriers associated with rare diseases

Thank you to Dr. Joanne Donovan, for taking the time to discuss the company’s innovative approach to targeting the “muscle as an organ” in its pursuit of treatments for rare neuromuscular and cardiac diseases, including Becker and Duchene muscular dystrophy.

Stay tuned for the next new episode of Rare Insights! In the meantime, listen to all our episodes Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “Rare Insights”.

Rare Insights’ team includes Taren Grom, Liz Kay, Kaitlyn Wosik, DNA Today’s Kira Dineen, and

myself, Nina Wachsman.

See what else we are up to on X, Instagram, Facebook, YouTube, and our website, knowrare.com. Questions/inquiries can be sent to [e-mail].