Proposed Mechanism of Action
ETF™ Gene Therapy*
- Recombinant hNGF genes are delivered into the lacrimal gland via non-replicating DNA delivery transporters1
- Endogenous cellular processes transcribe and translate the delivered genes into target proteins in the lacrimal gland1
- The proteins are transported into the tear film1
Endogenous hNGF in natural tear film
- Binds TrkA and p75NTR (high- and low-affinity hNGF receptors, respectively)2
- Involved in neuron differentiation and maintenance;3 required to maintain corneal sub-basal nerve architecture and corneal epithelial integrity4
- Basal tear film hNGF levels in healthy individuals: ~65 pg/mL5
The use of MR-146 for Neurotropic Keratopathy is currently being investigated and its safety and efficacy have not been established.
*ETF™ Gene Therapy is an investigational approach to therapy for ocular surface diseases that has not been tested in humans or approved for use in any country.1
DNA, deoxyribonucleic acid; ETF™, Enriched Tear Film; hNGF, human nerve growth factor; NK, neurotrophic keratopathy; p75NTR, p75 neurotrophin receptor; TrkA, tropomyosin receptor kinase A; VNS, OC-01 (varenicline solution) nasal spray.
1. https://www.ophthalmologytimes.com/view/oyster-point-pharma-unveils-preclinical-study-results-for-enriched-tear-film-gene-therapy-to-target-ocular-surface-diseases; 2. Reichardt LF. Philos Trans R Soc Lond B Biol Sci. 2006;361:1545–1564; 3. Martorana F. et al. Cell Death and Disease. 2018:391; 4. Shaheen BS. et al. Surv Ophthalmol vc. 2014;59:263–285; 5. Lambiase A. et al. Arch Ophthalmol. 2011;129:981-986.
*ETF™ Gene Therapy is an investigational approach to therapy for ocular surface diseases that has not been tested in humans or approved for use in any country.1
DNA, deoxyribonucleic acid; ETF™, Enriched Tear Film; hNGF, human nerve growth factor; NK, neurotrophic keratopathy; p75NTR, p75 neurotrophin receptor; TrkA, tropomyosin receptor kinase A; VNS, OC-01 (varenicline solution) nasal spray.
1. https://www.ophthalmologytimes.com/view/oyster-point-pharma-unveils-preclinical-study-results-for-enriched-tear-film-gene-therapy-to-target-ocular-surface-diseases; 2. Reichardt LF. Philos Trans R Soc Lond B Biol Sci. 2006;361:1545–1564; 3. Martorana F. et al. Cell Death and Disease. 2018:391; 4. Shaheen BS. et al. Surv Ophthalmol vc. 2014;59:263–285; 5. Lambiase A. et al. Arch Ophthalmol. 2011;129:981-986.