4DMT is building a pipeline of wholly-owned gene therapy products delivered by routine intravenous and / or intramuscular injection for patients with rare diseases involving skeletal muscles and other tissues. Intravenous and intramuscular injections are performed thousands of times daily in the US and the rest of the world to deliver treatments.
4DMT has a portfolio of optimized, proprietary and diverse AAV vectors for intravascular delivery to skeletal muscle and other tissues at doses that are orders of magnitude lower (vg/kg) than 1st and 2nd generation vectors. We start by defining the Target Product Profile for each skeletal muscle disease indication, and on that basis we can select the optimal customized vector from our portfolio of vectors. Our lead product for these patients (4D-510) will be administered by intravenous and/or intramuscular injection, and is designed to result in widespread expression of the therapeutic protein within cells in all major skeletal muscle groups and other target tissues. The target patient population for this product will be disclosed at the time of clinical trial initiation.