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 Inhaled mRNA May Help Rare Disease Patients Breathe Easier
Changing What’s Possible with Cell and Gene Therapies
Why Flying with a Wheelchair Is a Civil Rights Issue
Mapping Spheres of Influence
Bringing Precision to the Treatment of Rare Cancers
The Challenges of Developing a Therapy for Pregnant Women with A Rare Condition
How One Drug Developer Is Addressing Health Inequities
Accelerating the Development of Genetic Medicines with AI
Leveraging AI to Address the Neuropsychiatric Conditions Associated with a Rare Disease
In Pursuit of a Treatment for His Son, A Lawyer Becomes a Biotech Executive
In Push for Treatment, a Patient Organization Becomes a Trial Sponsor
Delayed Diagnosis of Rare Diseases Takes Staggering Economic Toll
A Cell Therapy to Control Heart-Damaging Inflammation in Duchenne
Stimulating the Growth of Muscle Mass in People with Neuromuscular Diseases
Turning Grief into Action
A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair Hearing
How the Woolly Mammoth Beat a Path to Better Gene Therapies
A Genetic Counselor’s Journey into Patient Advocacy
A Company in the Fold of Rare Disease
Disabling Antibodies that Drive Rare, Immunological Conditions
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