RARECast

Gaurav Shah, CEO of Rocket, discusses his company’s approach to gene therapy, the conditions it’s targeting, and how it determines what gene therapies it will pursue.

TSC Alliance CEO Kari Rosbeck and TSC Alliance Director of Medical Affairs Ashley Pounders, discuss the organization’s TSC Navigator, the thinking behind it, and why it could serve as a model for other patient organizations to follow.

Anurag Relan, chief medical officer of Pharming, discusses the rare immune condition APDS, the role PI3K inhibitors can play in treating it, and why a potential drug in development to treat the condition may have broader use in autoimmune and inflammatory diseases.

R.A. Session II, founder and CEO of Taysha Gene Therapies, discusses the origins of his company, its unique relationship with gene therapy innovator Steve Gray and UT Southwestern, and the critical role patient organization have played in the process.

Ken Mills, CEO of RegenxBio, discusses wet-age macular degeneration, the company’s collaboration with AbbVie, and the potential to alter the way patients with this and other eye conditions are treated by using a gene therapy that gets the body to produce a VEGF inhibitor.

Joe Truitt, CEO of iECURE, discusses his company’s in vivo gene editing therapies, its focus on liver diseases, and how it’s leveraging its partnership with the University of Pennsylvania’s Gene Therapy Program.

Josh Lehrer, CEO of Graphite Bio, discusses his company’s experimental sickle cell gene editing therapy, how it works, and what makes it a next-generation gene editor. @globalgenes #RARECast

Arthur Tzianabos, CEO of Homology Medicines, discusses the company’s genetic therapies, its program in PKU, and how it pairs its vectors and approach to meet the needs of a given condition. @globalgenes #RARECast

Jon and Shirley Dicks discuss the diagnostic odyssey for their daughter Elle, how having a diagnosis changed care for her, and the challenges they have faced caring for a child with a rare condition.

Nancy Yu, co-founder and CEO of AllStripes, discusses the growing efforts around the collection of patient data, where AllStripes fits into this emerging landscape, and how data can transform the outlook for diagnosing and treating people with rare diseases.