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.
How a Rare Diagnosis Impacts a Family
Transforming the Rare Disease Landscape with Data
Creating a Playbook for Bespoke Gene Therapies
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
How a Familiar Face Can Lead to a Rare Disease Diagnosis
Bringing the Trial to the Patient
Targeting Rare and Chronic Kidney Diseases
Addressing the Delivery Challenges of Genetic Medicines
Targeting Tissues Throughout the Body with RNA Therapies
Delivering Rare Disease Therapies to Patients in Need
An Advocate's Journey through Diagnosis, Loss, and Hope
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
How a Small and Young Foundation Catalyzed Research into a Rare Disease
Overcoming the Limitations of Conventional Cell Engineering
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
Forging Gene Therapy Capacity and a Pipeline at the Same Time
Using A Natural DNA Repair Process to Improve Genetic Medicines
Empowering Rare Disease Patients with their Own Health Records
Targeting CNS Disease with Gene Therapies
A New Therapy Offers A Different Approach to Inhibiting the Complement System
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