Pipeline

The GENETIX pipeline includes:

  • LentiVue™ for macular degeneration -- localized ophthalmic delivery (academic collaboration with Columbia University)
    • Stargardt, Usher genetic diseases
    • Age-related macular degeneration
  • LentiNeur™ for neurological disorders -- localized stereotactic delivery for Parkinson’s disease
  • LentiRA™ for rheumatoid arthritis -- localized to major joints (academic collaboration with Harvard University / Brigham & Women's Hospital)
  • LentiFVIII™ for hemophilia A -- Factor VIII gene delivery to blood stem cells (GENETIX SBIR)
  • LentiDL™ for familial hypercholesterolemia -- LDL receptor gene therapy localized to liver (academic collaboration with INSERM / Hospital of Paris)
  • Further genetic disorders -- cellular delivery for SCID, CGD, Adrenoleukodystrophy, Gaucher Disease, others

 

 

 

 

LentiPak™

The LentiPak™ gene delivery platform is an exceptionally efficient approach to gene delivery, typically resulting in transfer and expression of a therapeutic gene in a majority of treated animal or human stem cells. Lentiviruses are capable of transferring genes to the genome of non-dividing cells as well as dividing cells, making LentiPak™ gene delivery particularly well-suited for stem cell gene therapy because stem cells divide infrequently. Since lentiviruses retain their superior capability for gene transfer, LentiPak™ represents a promising platform technology for delivery of therapeutic genes potentially useful for the treatment of a wide variety of human diseases.