Purpose

To create highly effective 3rd-generation AAV gene therapy products from our Directed Vector Evolution discovery platform to cure patients with severe rare genetic diseases.

 

GUIDING PRINCIPLES

 

  1. Innovate to create better gene therapy products
  2. Invent optimized vectors that are the “gold standards” for the field
  3. Develop and commercialize our own wholly-owned products for rare diseases
  4. Grow a diverse pipeline of products for retina, heart, muscle and other therapeutic areas
  5. Partner with top Biopharmaceutical Companies with complementary capabilities to
    maximize product development efficiency
  6. Partner with leading Patient Advocacy Organizations to maximize patient benefit

Mission

  • To translate gene therapy products into clinical development and commercialization for the retina, heart, muscle tissues and other therapeutic areas

  • To demonstrate 4DMT vector and product superiority over other AAV gene therapy products in clinical trials in diverse patient populations

  • To discover, manufacture and patent highly optimized AAV vectors for clinical delivery to at least 10 important human tissues (achieved)

Vision

Our company’s vision is to be a world leader in AAV gene therapy. This means creating and continually growing a transformative, valuable and diverse product pipeline and marketed product portfolio. So how do we measure value? Quite simply, we base the value of our pipeline and portfolio on three features:

1. How many different debilitating diseases do our products fight? We plan to battle more diseases, in more tissue types, than any other organization.

 

2. How many individual patients live better and longer lives because they’ve been treated with our products? We will never stop bringing new products to patients who need them.

3. How well do our products work? We are striving to create truly transformative products that can lead to cures. Patients should live markedly better and longer lives because they received treatment with a 4DMT product.