Rare Insights: Uncovering The Future Of Rare Disease Treatments

#1 Sustaining A Rare Disease Pipeline with Rocket Pharma

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

Introducing Rare Insights

“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.
 
Be sure to follow us on social media! We are @KnowRare on Instagram, X, and LinkedIn. 
 
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.