GENE THERAPY MODELS
Validated and reliable models for ocular gene therapy.
One of the main reasons that the eye is a great target for gene therapy is the fact that some compartments in it lack direct connections to any sort of vessels, making it one of the few “immune-privileged” organs in the human body alongside the CNS and the testes. Out of all gene therapy clinical trials ever run, roughly 1.3% are for ocular diseases, a small number when the advantages that the eye has as a gene therapy target are factored in.
Advances in understanding the pathogenesis of ocular diseases has paved the way for the development of different kinds of gene therapies, including both gene silencing and replacement, both of which are promising. Your pipeline along with our validated, reliable and carefully designed models are all it takes to find the next treatment, and maybe even cure, for another ocular disease.
MODELS / SERVICES
Extensively validated preclinical models:
- Scientific staff with an average of over 15 years of experience in pharmacology
- 600+ validated animal models of disease spanning 40+ diseases
- 150,000 square feet of facility space
- 24/7 access to PharmaLegacy representatives