It's In The Genes: A Gene Therapy Podcast

As gene therapy research becomes a reality for how we view and treat medicine, we ask our researchers why they devoted their professional lives to this science. In our season finale episode of It’s in the Genes, we will hear from a variety of researchers, scientists, and experts to better understand why they are so committed to investigational gene therapy and working towards ensuring protected, purposeful and progressive science moves forward in the future. Featuring Dr. Glenn Pierce, Dr. Sven Kili, Dr. Theodore Freidmann, and many more! Program Notes: Want to discover more about gene therapy research? Subscribe here (GT Pod link) BloodStream Journeys is exclusively sponsored by BioMarin: www.biomarin.com Visit HemDifferently at https://www.hemdifferently.com/ for more educational information on gene therapy research! Connect with BloodStream Media: Find all of our bleeding disorders podcasts on BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter Check out Believe Limited’s Other Work: BloodFeed: bloodfeed.com Bombardier Blood: bombardierblood.com Hemophilia: The Musical: breakingthroughhemophilia.com My Beautiful Stutter: mybeautifulstutter.com/ Stop The Bleeding!: stbhemo.com Teen Impact Awards: teenimpactawards.com The Science Fair: thesciencefair.org

What does the decision-making process look like for a patient considering a gene therapy trial? In episode four of It’s in the Genes, we’ll hear from a patient who is currently experiencing a gene therapy research trial. We’ll learn why he committed to this method of treatment and how it has impacted him. Join us as he shares his experiences, the advice he gives to the other patients considering participating in a trial, and how the experience challenged him both physically and mentally. Program Notes: Want to discover more about gene therapy research? Subscribe here (GT Pod link) BloodStream Journeys is exclusively sponsored by BioMarin: www.biomarin.com Visit HemDifferently at https://www.hemdifferently.com/ for more educational information on gene therapy research! Connect with BloodStream Media: Find all of our bleeding disorders podcasts on BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter Check out Believe Limited’s Other Work: BloodFeed: bloodfeed.com Bombardier Blood: bombardierblood.com Hemophilia: The Musical: breakingthroughhemophilia.com My Beautiful Stutter: mybeautifulstutter.com/ Stop The Bleeding!: stbhemo.com Teen Impact Awards: teenimpactawards.com The Science Fair: thesciencefair.org

Clinical trials are the process of designing a controlled experiment to test drugs in humans, which has taken decades to shape and perfect! In episode three of It’s in the Genes, we dive into the clinical trial process for investigational gene therapy with Boston Children’s gene therapy research nurse, Colleen Dansereau. Clinical trials are crucial to the process and the data collected can have an impact on the future. Join us to learn more about clinical trial procedures! Program Notes: Want to discover more about gene therapy research? Subscribe here BloodStream Journeys is exclusively sponsored by BioMarin: www.biomarin.com Visit HemDifferently at https://www.hemdifferently.com/ for more educational information on gene therapy research! Connect with BloodStream Media: Find all of our bleeding disorders podcasts on BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter Check out Believe Limited’s Other Work: BloodFeed: bloodfeed.com Bombardier Blood: bombardierblood.com Hemophilia: The Musical: breakingthroughhemophilia.com My Beautiful Stutter: mybeautifulstutter.com/ Stop The Bleeding!: stbhemo.com Teen Impact Awards: teenimpactawards.com The Science Fair: thesciencefair.org

Gene therapy research is complex science! Such complex science is sure to bring setbacks and challenges to overcome. In episode two of It’s In The Genes, listen in as our experts share how gene therapy research has been conducted, the roadblocks investigational gene therapy researchers faced, and how they accounted for safety in the process. Featuring prominent gene therapy researchers, Dr. Theodore Friedmann, Dr. Richard Samulski, Dr. Jennifer Adair, and Dr. Sven Kili. Program Notes: Want to discover more about gene therapy research? Subscribe here (GT Pod link) BloodStream Journeys is exclusively sponsored by BioMarin: www.biomarin.com Visit HemDifferently at https://www.hemdifferently.com/ for more educational information on gene therapy research! Connect with BloodStream Media: Find all of our bleeding disorders podcasts on BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter Check out Believe Limited’s Other Work: BloodFeed: bloodfeed.com Bombardier Blood: bombardierblood.com Hemophilia: The Musical: breakingthroughhemophilia.com My Beautiful Stutter: mybeautifulstutter.com/ Stop The Bleeding!: stbhemo.com Teen Impact Awards: teenimpactawards.com The Science Fair: thesciencefair.org

Where did the idea of gene therapy research originate from and what was it like at the forefront of scientific discovery? Join us for episode one of It’s In the Genes: A Gene Therapy Podcast as we journey into the history of how investigational gene therapy came about, the twists and turns the field found themselves in, and the implications of such innovative science. Featuring gene therapy research leaders, Dr. Theodore Friedmann and Dr. Richard Samulski. Program Notes: Want to discover more about gene therapy research? Subscribe here (GT Pod link) BloodStream Journeys is exclusively sponsored by BioMarin: www.biomarin.com Visit https://www.hemdifferently.com/ for more educational information on gene therapy research Connect with BloodStream Media: Find all of our bleeding disorders podcasts on BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter Check out Believe Limited’s Other Work: BloodFeed: bloodfeed.com Bombardier Blood: bombardierblood.com Hemophilia: The Musical: breakingthroughhemophilia.com My Beautiful Stutter: mybeautifulstutter.com/ Stop The Bleeding!: stbhemo.com Teen Impact Awards: teenimpactawards.com The Science Fair: thesciencefair.org

On August 5th join us for a journey into the complexity and mystery of gene therapy research! Over 5 episodes, we’ll discuss the triumphs and setbacks that shaped the revelation of gene therapies from the brilliant minds that were at the forefront of discovery. Hear from Dr. Theodore Friedmann and Dr. Richard Samulski share stories, pivotal moments, and chance meetings that will come to define the new age of rare disease treatment! Program Notes: Want to discover more about gene therapy research? Subscribe here! It’s In The Genes is exclusively sponsored by BioMarin: www.biomarin.com Visit https://www.hemdifferently.com/ for more educational information on gene therapy research Connect with BloodStream Media: Find all of our bleeding disorders podcasts on BloodStreamMedia.com BloodStream on Facebook BloodStream on Twitter Check out Believe Limited’s Other Work: BloodFeed: bloodfeed.com Bombardier Blood: bombardierblood.com Hemophilia: The Musical: breakingthroughhemophilia.com My Beautiful Stutter: mybeautifulstutter.com/ Stop The Bleeding!: stbhemo.com Teen Impact Awards: teenimpactawards.com The Science Fair: thesciencefair.org