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.
Bringing Aberrant Proteins Back into the Fold
A Cat who Curates Rare Mice
Putting the Patient at the Center of Rare Disease Clinical Trials
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
PTC Looks to Advanced Therapies
Going to Extreme Lengths
Everything’s up to Date in Kansas City, at Least When It Comes to Genomics
Choosing the Right Viral Vector for a Gene Therapy
A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases
Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure
Realizing the Potential of CRISPR Gene Editing
Understanding the Economic Toll of Rare Disease in the United States
The Promise of Gene-Based Therapies for Neurodegenerative Conditions
Moving Beyond Viral Vectors for Gene Therapies
Acquisitions Help Jazz Build Toward Commercial Crescendo
Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases
A Child's-Eye-View of Clinical Trials
Using Nanoliposomes to Make Cancer Therapies Safer and More Effective
Accelerating Treatments for Rare Disease through Data Sharing
Treating the Root Cause of Sickle Cell Disease
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