TARGTā„¢
(Transduced Autologous Restorative Gene Therapy)

      1. Harvest Micro-Organs from abdominal dermis using the proprietary DermaVac device
      2. Transduce fibroblasts in Micro-Organs via ex vivo gene therapy to create TARGTEPO
      3. Wash out HDAd vector, measure production and choose TARGTEPO for re-implantation
      4. Implant TARGTEPO into patient where it produces endogenous EPO (eEPO)

 

Key Advantages of TARGT:

Continuous, autologous protein production that offers several advantages over recombinant protein therapies:

 

Improved safety, tolerability,
and compliance:

 

Capital efficient, de-risked
path to drug development:

  • Closely mimics natural system, creating restorative treatment paradigm
  • Improved durability in vivo
  • Greater cell uptake
  • Less immunogenicity
 
  • Ex vivo transduction eliminates patient exposure to viral vectors typically seen in gene therapy
  • Reduced chance of developing neutralizing antibodies which can lead to reduced efficacy and anaphylactic reactions
  • Ability to control dosing through the addition and removal of micro-organs as needed
  • Implantation ensures compliance and better outcomes, and provides control of the product
 
  • Exogenous gene therapy on “tummy tuck” tissue is performed to test human protein expression in vitro
  • Severe combined immunodeficient (SCID) mice provide predictive in vivo model for protein expression and duration
  • In vitro (human) and in vivo (SCID mouse) models provide
    pre-clinical POC, for approximately $100K and less than 6 months
  • Platform eliminates the need for large investment in manufacturing and scale up for clinical trial material