RARECast RARECast

Doron Behar, co-founder and CEO of Igentify, discusses the company’s AI-powered Digital Genetic Assistant, how it works, and why it will allow genetic counselors to handle a much larger volume of patients.

Frank Gleeson, co-founder and CEO of Satellos, discusses its regenerative therapy for Duchenne, how it works, and why it may provide benefits to patients with other conditions that result in muscle degeneration.

Courtney Silverthorn, vice president of strategic alliances and innovation for the Foundation for the National Institutes of Health; and Sharon King, manager of advocacy and community engagement for Aldevron and founder and president of Taylor’s Tale; discuss the Bespoke Gene Therapy Consortium, its new playbook, and how it will help gene therapy developers get their medicines to patients faster.

Rick Chapman, chief scientific officer of the Innovation and Value Initiative, discusses the challenges of assessing the value of rare disease therapies, the role qualitative data should play in value assessments, and the recommendations from the report.

Ha Tran, medical head of cell and gene therapy for Astellas Pharma, discusses the company’s vision for cell and gene therapies, its efforts to restart trials for its X-linked myotubular myopathy gene therapy, and how it is looking to other programs beyond that.

Neal Sondheimer, outgoing head of clinical for Synlogic and adjunct associate professor of pediatrics and molecular genetics for The Hospital for Sick Children in Toronto, discusses PKU, the treatment options for people with the condition, and the consequences of the surprising results from the Synlogic study.