Rare Disease and a Repeatable Process

Of all the presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, a presentation by renowned physician and geneticist Dr. Francis Collins caught our attention. In his talk, Dr. Collins spotlighted an encouraging path forward in the treatment of rare diseases with cell and gene therapies. While acknowledging that gene therapy is being taken to new heights in many areas, he noted there are still thousands of diseases that have not yet had a focus for treatment development.

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