In this presentation, Dr. Gray will describe how gene therapies have emerged as a powerful therapeutic modality for treating a range of genetic diseases. Drawing from his experience designing clinical gene therapy vectors and the manufacturing systems used to generate clinical-grade products, he will review the major scientific challenges to efficient therapeutic delivery of genes to patients’ cells. Data from successful clinical trials of genetic therapies for immunodeficiency, hemophilia, and myotubular myopathy will be presented to illustrate how gene delivery technology has evolved to meet many of these challenges. The potential to leverage these successes into therapeutic gene delivery platforms will be discussed in the context of the remaining scientific challenges and the need to develop therapies for more challenging diseases with severe unmet medical need.
The IGI seminar series features a diverse range of speakers focusing on a variety of topics related to the Institute’s research programs. Following all seminars, attendees are invited to stay for a free lunch and talk with the seminar speaker.
From Blood to Liver, Muscle, and Beyond:
Delivering Genes Where They Are Needed Most
Dr. John Gray
Senior Vice President, Chief Scientific Officer
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