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.
Pregnancy and Motherhood for a Woman with SMA
Steps to Improve Data Gathering of Rare Diseases
Rare Patients Learn to Make Their Voices Heard
Empowering Patient Organizations with Real-World Data
A Healthcare Communications Student Gets an Education as a Patient
Expanding the CRISPR Toolkit
Marrying Antibodies to RNA Therapies to Target Previously Inaccessible Tissues and Cells
Expanding Access to Whole Genome Sequencing Across the Globe
A First-in-Class Approach to Treating a Rare and Chronic Liver Disease
A New Approach to Treating a Rare Endocrine Disorder
Detecting Genetic Disease Prior to Birth
Turning Words into Action: Equity, Diversity, and Inclusion in Rare Disease
Chasing Cures
A Map to Navigate A Rare Disease
A Popular Cancer Target Is Eyed for a Rare Immune Disorder
Leveraging a Gene Therapy Approach to Maximize Speed and Minimize Costs
Programing the Body to Make Its Own Medicine with Gene Therapy
Developing in Vivo Gene Editors that Target Liver Diseases
A Find-and-Replace Approach to Fixing the Mutation Underlying Sickle Cell Disease
A Bet on a Unique Set of Vectors
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