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Alan Cohen, senior vice president of clinical development and therapeutic area head of pulmonology for 4DMT, discusses the limitations of existing vectors for genetic medicines, 4DMT’s directed evolution platform technology, and its programs in cystic fibrosis and Fabry disease.

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.