SmartPharm’s gene-encoded therapeutics (GET) approach has multiple benefits compared to traditional viral-vectored gene therapy:

  • Minimally immunogenic; does not require immune suppression

  • Re-dosable in patients

  • Scalable, economic cGMP manufacturing

  • Reduced regulatory complexity

Other non-viral GET companies use either DNA or mRNA approaches. SmartPharm’s platform technology includes both technologies so we can address a broad range of therapeutic opportunities.

  • Selects type of nucleic acid based on specific therapeutic target and purpose

  • Protein sequence tuned for use with the specific nucleic acid platform and for the specific therapeutic application

Our Focus

  • Eliminating the need for recombinant enzyme replacement therapy in lysosomal storage diseases with a first drug candidate in Gaucher disease type 1

  • New treatments for tissue restoration and regeneration

  • Cancer biologics “biobetters”

Our Culture and Leadership

Working at SmartPharm