We are creating the ultimate gene therapy products to cure genetic diseases.
Our company’s vision is to be the world leader in gene therapy. This means creating and continually growing the most valuable product pipeline and marketed product portfolio in the industry. This is the 4D value proposition.
So how do we measure “value”? Quite simply, we base the value of our pipeline and portfolio on three features:
- First, what is the number and diversity of the debilitating diseases our products fight? We plan to battle more diseases, in more tissue types, than any other organization.
- Second, how many individual patients live better lives because they’ve been treated with our products? We are targeting both monogenic hereditary disorders and complex large market diseases. We will never stop bringing new products to patients who need them.
- Third, how well do our products work? We are striving to create truly transformative products that can lead to cures. Patients should live markedly better lives because they received treatment with a 4D product.
To create the ultimate gene therapy products from our Directed Vector Evolution discovery platform to cure patients with severe genetic diseases.
To discover and patent novel AAV vectors for clinical delivery to 10 different human tissues with the following attributes:
- Order of magnitude improvement in gene delivery and expression versus standard first-generation vectors
- Efficient delivery despite pre-existing immunity to natural AAV serotypes in the general population
- Efficient delivery by standard clinical routes that will maximize infection of target tissues
- Strong manufacturing scalability
- Strong composition-of-matter patentability