Imagine walking into a hospital with your child who has an unknown disease. A nurse takes a DNA sample and in days identifies the disease’s genetic cause. Expert engineers construct a vector to replace the mutated gene, and manufacturing is mobilized to produce it, generating a life-saving treatment in months. Believe it or not, we are entering that era now. Expert gene therapists are churning out potential therapies for devastating diseases, and companies are building the manufacturing infrastructure to bring the therapies to those in need. In this panel, a scientist on the frontiers of gene therapy, the executive driving the development to make these therapies a reality for all and a parent actively searching for a therapy will discuss the uncharted territory that is gene therapy.
Other Resources / Information
- The promise of gene therapy has been realized and development will accelerate in near future.
- Gene therapy can help millions of children who otherwise would only have palliative care to manager their disease.
- The strongest programs will rely on close interaction with patient and parent communities.
- RA Session, Founder, president and CEO, Taysha Gene Therapies
- Steven Gray, Associate Professor, University of Texas Southwestern Medical Center
- Sayak Mukherjee, Writer, Fortune Magazine
- Kasey Woleben, Founder, Cure SURF1 Foundation and Rare Village Foundation
Carolyn Hawley, Vice President, Canale Communications
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