Joys of Compounding
Episode 7 Compounding For A Cure
Joys of Compounding

Episode 7: Compounding For A Cure

Compounding For A Cure

Jeff Marrazzo is the Co-Founder of Spark Therapeutics. We cover the uncommon sources of inspiration that led to the founding of Spark, how it has the potential to revolutionize gene therapy, and lessons learned from navigating the complex world of FDA approval.

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(00:06:20) - [First question] - Witnessing the impact of aiding others

(00:10:45) - The genesis of the vision for Spark Theraputics

(00:15:11) - Securing FDA approval

(00:20:22) - Navigating the venture capital landscape

(00:27:26) - Confronting periods of doubt

(00:33:21) - The significance of gene therapy in the US

(00:38:40) - Drawing inspiration for interdisciplinary collaboration

(00:43:58) - Guidance for youth seeking to capitalize on opportunities

(00:50:20) - Anticipating advancements in life sciences and healthcare

(00:54:34) - The role of AI in advancing gene therapy

(00:58:38) - Ways to engage with biotechnology

(01:04:55) - Simplifying the complexities of biotechnology investment

 

Compounding For A Cure

Introduction

Rick
Our teacher today is giving sight to the blind. And no, I'm not talking about Jesus Christ, although he's always most welcome in our classroom. I'm talking about Jeff Marrazzo, Cofounder and former CEO of Spark Therapeutics, the biotech company that under Jeff's leadership was the very first in history to receive FDA approval for a gene therapy.

They did so with the drug LUXTURNA to treat inherited retinal disease. Unlike any treatment that came before it, LUXTURNA is a onetime gene therapy that has the potential to actually restore the visual cycle, the process that allows you to see. It's hard to overstate how revolutionary this breakthrough was not just for patients suffering from various IRDs but for the entire biotech complex as Spark has helped to ignite and expand the art of what's possible and how we will seek to treat and potentially cure hundreds of genetic conditions over the coming decades.

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