The Bespoke Gene Therapy Consortium, a public-private partnership backed by the Foundation for the National Institutes of Health, in February published its first playbook. The playbook provides a roadmap for streamlining product development and navigation of the regulatory pathway for AAV gene therapies with the goal of getting new treatments to patients sooner. We spoke to 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; about the Bespoke Gene Therapy Consortium, its new playbook, and how it will help gene therapy developers get their medicines to patients faster.
Restoring Hearing through Gene Therapies
Creating a Sustainable Reimbursement Model for Ultra-Rare Therapies
Lumos Hopes Its Oral Alternative to HGH Will Fuel Its Growth
Advancing a Gene Therapy for a Rare and Fatal CNS Disorder
Addressing the Barriers to Patient Participation in Clinical Trials
A Venture Philanthropist Makes the Case for Advocates Taking Equity
Seeking a Sustainable Business Developing on N-of-1 Therapies
How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition
A Next-Generation RNA Therapy Targets Telomere Disorders
BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families
Translating Urgency into Science
Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments
Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X
An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty
Powering Cells in People with Rare Mitochondrial Diseases
Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy
Developing a New Class of Therapies Based on a Natural Cargo Carrier
Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies
One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia
Treating Rare Endocrine Disorders with Therapeutic Peptides
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