NIH Gene Therapy Symposium

September 26-27, 2011
Natcher Conference Facility, National Institutes of Health
Bethesda, Maryland, USA

There are several initiatives that have come out of the NIH Gene Therapy Symposium, including:

  • Communication of outcomes to ASGCT Membership
  • Letter to Francis Collins detailing the outcomes and next steps for ASGCT,  including the identification of the ten target disease most likely to have a validated therapeutic in the next five to seven years
  • Formulating a panel of ASGCT members to help guide the Office of Rare Disease Research in the grouping of rare diseases into categories of diseases with common clinical phenotypes
  • Formulating a panel of ASGCT Members to engage in a dialogue with the RAC regarding ways to streamline the current regulatory process for gene therapists

"Target 10" Letter to Dr. Francis Collins

Dr. Barrie J. Carter - NIH History [YouTube]

Read the recently published article from Science Magazine (October 2011) on the successes of gene and cell therapy:


Last Updated January 19, 2012

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