Our Mission Statement:

  1. to bring therapies for rare diseases, involving viral vector delivery of DNA to the CNS by various routes, from late-pre-clinical development through to human trials and to public availability as efficiently as possible.
  2. to conduct our work with scientific integrity in data interpretation and fiducial transparency in our operations, and to make all data available to those willing to help develop therapies to the point of approval by the FDA and regulatory agencies and into medical practice, to the extent allowed by law.
  3. to select only therapies for diseases that have a reasonable chance of providing meaningful clinical benefits to patients; to raise and use only the funds needed to accomplish our stated objectives, sustain our organization’s continuation, and accomplish its mission; and not to engage in basic or exploratory research better accomplished by academic laboratories or others.
  4. to be good stewards of the worth of our employees, and their need for meaningful work and satisfying use of their talents and abilities.
  5. to communicate clearly and frequently with the families, foundations, government agencies, and others who support and enable our work.