Pipeline & Clinical Trials

Pipeline & Clinical Trials

Our Pipeline

We are developing a robust pipeline of disease-modifying therapies for orphan conditions with high unmet medical needs.

Fibroblasts, the most common cell in skin and connective tissue, repair tissue infrastructure by producing extracellular matrix proteins, including collagen. Our proprietary, autologous fibroblast technology platform is the basis of our cell-based, gene therapy pipeline.

Advantages of Our Approach

The use of autologous fibroblasts in our proprietary technology platform provides distinct advantages: for developing novel personalized treatments.

Fibroblasts are an ideal delivery vehicle to administer the protein of interest directly at the site of action:

  • Localized administration avoids side effects typically associated with systemic therapy
  • Reduced risk of rejection or immunogenicity because autologous fibroblasts are compatible with the unique biology of each patient

Use of a third generation, self-inactivating (SIN) lentivirus:

  • Accommodates large gene constructs
  • Allows for target gene integration, ideal for long-term expression of the protein of interest

Fibroblasts are genetically-modified ex vivo—providing safety and efficacy advantages:

  • Enables testing for safety and confirmation of transduction rates and protein expression prior to administration
  • Reduces potential for host immune response upon retreatment as live virus is not administered directly into the patient