#1 Sustaining A Rare Disease Pipeline with Rocket Pharma

Rare Insights: Uncovering The Future Of Rare Disease Treatments

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.

2. 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.

3. 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.

4. 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.

5. 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 [e-mail].