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 podcast@knowrare.com.

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:

1. Advancing CAP-1002 for Duchenne Muscular Dystrophy: Dr. Marbán talks about the progress of CAP-1002 entering Phase 3 trials and the next steps towards FDA approval.
2. Unique Approach with Exosome Technology: Exploring how Capricor’s use of exosome technology differentiates their treatment for Duchenne muscular dystrophy from other drugs in development.
3. Future of Cell and Exosome-Based Therapeutics: Insights into the potential of these innovative therapies to transform the treatment landscape for various rare diseases.
4. Shifts in the Rare Disease Space: Reflecting on major changes in the rare disease sector since co-founding Capricor in 2005 and the current trends that Dr. Marban is monitoring.
5. Commitment to the Rare Disease Community: Dr. Marbán shares what drew her to the rare disease field, her career-long dedication, and her passion for working with this unique community of patients and caregivers.

Dr. Marbán is currently serving as CEO, and has served in that capacity and on the Board since November 2013. As co-founder of Capricor, Inc., our wholly-owned subsidiary, Dr. Marbán has been with Capricor, Inc. since 2005 and became its CEO in 2010. Dr. Marbán has been in the biotechnology field for more than 20 years and brings extensive experience across research, product development and business development to the company.

From 2003-2009, Dr. Marbán held various senior roles at Excigen, Inc., a gene therapy biotechnology company, where she was responsible for operations and business development and where she oversaw the development of a biologic pacemaker for the heart. Prior to Excigen, Dr. Marbán worked in academic science, first at the Cleveland Clinic Foundation working on the development of contractile dysfunction in heart failure due to myocarditis, followed by a postdoctoral fellowship at Johns Hopkins University. While at Johns Hopkins, she advanced to the rank of Research Assistant Professor in the Department of Pediatrics, specializing in the mechanism of the biophysical properties of cardiac muscle. Her tenure at Johns Hopkins ran from 2000 to 2003.

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 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 podcast@knowrare.com.

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 disease drug development and the unique aspects that draw professionals to this field.

Discussion Topics:

1. Introduction to Ascidian’s Technology: Exploring the innovative RNA editing inspired by "sea squirts" and its implications for treating rare diseases.
2. Reducing Risks in Gene Editing: How Ascidian’s RNA exon editing approach minimizes risks compared to traditional gene editing methods.
3. FDA Fast-Track Designation for ACDN-01: Insights on achieving fast-track status for Stargardt disease treatment and future plans for Ascidian’s pipeline.
4. RNA Editing Momentum: The pressures and focus brought by the increasing interest in RNA editing.
5. Personal Journey in Rare Diseases: Dr. Barth shares what inspired his career in the rare disease sector and what continues to excite him about this work.

Dr. Jay Barth is Chief Medical Officer of Ascidian Therapeutics. With 25 years of experience in drug development and medical affairs, and a focus in gene therapy and rare diseases, Dr. Barth will be responsible for all clinical development activities across Ascidian's diversified pipeline in ophthalmology, neurological, neuromuscular, and genetically defined diseases. Dr. Barth has wide-ranging clinical development expertise across many challenging therapeutic categories. He led the clinical efforts of the Marketing Authorization Application (MAA) process leading to the first regulatory approval of a treatment for Duchenne muscular dystrophy while at PTC Therapeutics. As CMO at Amicus Therapeutics, he oversaw the clinical components of the EU marketing application which culminated in the first oral treatment of Fabry disease. Dr. Barth has also worked extensively with regulatory affairs teams in the U.S. and EU to develop regulatory strategies, liaise with regulatory agencies, and prepare marketing applications.

Early in his career, Dr. Barth developed drugs for broad patient populations, primarily in the areas of gastrointestinal and pulmonary diseases, at companies including Eisai Medical Research and Merck. His subsequent drug development work shifted to rare diseases and then specifically to gene therapy. Immediately prior to joining Ascidian, Dr. Barth served as CMO for Lexeo Therapeutics, a clinical-stage gene therapy company focused on addressing cardiovascular and central nervous system diseases. At Lexeo, he led the team's efforts in the first IND cleared by the FDA for a gene therapy targeting Friedreich's ataxia cardiomyopathy.

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 Taylor, DNA Today’s Kira Dineen, and 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 podcast@knowrare.com.

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 leadership team with oversight spanning discovery through late-stage development and manufacturing. This includes regulatory, clinical, global program teams, CMC, quality and the development organization. Dr. Patel’s prior responsibilities at Rocket included IR, IT, HR, finance and corporate compliance. Dr. Patel has more than 20 years of R&D experience in rare diseases and across several therapeutic areas including immuno-oncology, oncology, respiratory, virology, transplantation, cardiology and metabolism.

Discussion Topics:

1. Advancing the company’s six rare disease assets: Dr. Patel provides an update on Rocket’s pipeline.

1. Update on Kresladi: Insights into marnetegragene autotemcel’s progress from IND to BLA for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I) in fewer than five years.

1. Bringing down the risk of drug development: Dr. Patel reflects on how the company’s experience and operational expertise of taking a platform portfolio approach is helping to advance the pipeline.

1. A culture of patient focus: Building trusted relationships with everyone involved, learning how to be generous with knowledge, and being curious are keys to Rocket’s success.

1. The rare disease space looking forward: Dr. Patel identifies several trends she hopes will impact patients — doubling or tripling the number of gene therapies approved, bringing down the cost of developing gene therapies, and making rare disease therapies available to communities around the world.

Stay tuned for the next episode of Rare Insights in July! 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 podcast@knowrare.com.

“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 from passionate industry leaders. Together, we navigate the unknowns and unlock the potential for groundbreaking treatments. Because in this journey, we're all rare, sharing unique insights and experiences that drive innovation and progress.

Our host is Taren Grom, a well-respected media executive, who co-founded and then sold PharmaVoice, the preeminent trade magazine for life science executives at all stages of their careers. PharmaVoice focused on humanizing the people behind the science, a similar sensibility to Know Rare.

If you are in the biopharma industry and would like more information about Know Rare, visit knowrare.com/sponsors. Any questions, episode ideas, guest pitches, or comments can be sent into podcast@knowrare.com. For our blog and other podcasts and video content, visit knowrare.com.

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Please rate and review the podcast on Apple, Spotify, or wherever you listen. This truly helps us climb the charts and allow more bio-pharmaceutical executives and other industry leaders, like yourself, to discover the show.

Rare Insights’ team includes Taren Grom, Liz Kay, Kaitlyn Taylor, DNA Today’s Kira Dineen, and Nina Wachsman, CEO and co-founder of Know Rare.

We hope you enjoy listening to Rare Insights. Join us to uncover the future of rare disease treatment.