03 May Caeregen Therapeutics Announces Data Presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting
- Data presented on promising activity of Noregen™, first potential regenerative therapy for retinal diseases
- Follows 1Q2022 U.S. FDA and EU EMA Orphan Drug Designation (ODD) for Noregen™ in Familial Exudative Vitreoretinopathy (FEVR)
May 3, 2022, Denver, CO and Rochester, MI – Caeregen Therapeutics, LLC, a Michigan-based, regenerative medicine therapeutics company today announced data on Noregen™ from studies conducted with the Oakland University Eye Research Institute (ERI), at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting in Denver, CO.
The poster titled “Development of Noregen™, A Novel Regenerative Ocular Therapeutic Medicine” indicated that in the pre-clinical in vivo model of oxygen induced retinopathy (OIR), Noregen demonstrated statistically significant (p=0.027) regeneration of degraded retinal vasculature. Further, Noregen demonstrated statistically superior (p=0.0001) in vitro proliferation of Human Retinal Microvascular Endothelial Cells (HRMEC), validated through gene expression assays revealing 2-fold increase in Axin-2 (Wnt-targeted cell growth) signaling and >50% reduction in Plasmalemma Vesicle Associated Protein (PLVAP) expression, revealing the barrier stabilizing character of Noregen. Importantly, the safety of Noregen was also demonstrated through in vivo assessments of edema, ERG response and microvasculature.
“The pre-clinical, in vivo validation of Noregen’s promising regenerative properties for the retina, first seen through in vitro testing, was especially gratifying” said Kimberley Drenser, M.D., Ph.D., co-founder and Chief Scientific Officer of Caeregen Therapeutics. “We look forward to advancing Noregen’s development in the pursuit of improving clinical outcomes for patients at risk of progressive, retina-related vision loss” added Dr. Drenser.
Noregen is a synthetic targeted growth-factor, developed by Caeregen, that mimics the properties of norrin, a naturally occurring human protein that promotes development of normal, organized blood vessels and neurons in the human eye, ear, and central nervous system. Noregen is being developed for Familial Exudative Vitreoretinopathy (FEVR), a rare genetic disorder of retinal blood vessel formation, resulting in partial or complete vision loss. When injected into the eye, Noregen may regenerate retinal blood vessels and neurons restoring normal retinal function and counteracting vision loss due to FEVR. Vision-threatening retinal diseases such as retinal vein occlusion (RVO) and diabetic retinopathy (DR) are further areas of urgent unmet patient need and are priorities for Noregen development.
“Obtaining such encouraging pre-clinical results with Noregen, coming so shortly after obtaining Orphan Drug Designation from both the US FDA and EU EMA in the indication of FEVR, is significant milestone for patients” said Michael T. Trese, M.D., co-Founder and Chief Technology Officer of Caeregen Therapeutics. “As a novel regenerative medicine, Noregen has the potential to be the first treatment for FEVR to actually re-activate growth of normal retinal blood vessels and neuronal cells, in order to restore and maintain vision” added Dr. Trese.
Noregen™ (CT-107) is a synthetic targeted growth-factor developed by Caeregen Therapeutics for retina-related vision loss modeled after norrin, a naturally occurring human protein that guides retinal formation in fetal development. Noregen™ may promote development of normal, organized blood vessels and neurons in the human eye, ear, and central nervous system for individuals with inherited or acquired retinal diseases and potential applications in other neurosensory diseases. As a first-in-class therapeutic candidate to regenerate and repair the retinal vasculature in order to restore and preserve vision in patients with ischemic vitreoretinopathies, Noregen™ is currently being developed for Familial Exudative Vitreoretinopathy (FEVR), a rare disease of urgent, unmet medical need. Further applications for Noregen™ development include retinal vein occlusion (RVO) and diabetic retinopathy (DR). For more information, please visit: www.caeregen.com.
About FAMILIAL EXUDATIVE VITREORETINOPATHY (FEVR)
Familial Exudative Vitreoretinopathy or ‘FEVR’ is a rare inherited disorder of retinal angiogenesis, leading to incomplete vascularization of the peripheral retinal and poor vascular differentiation. FEVR most often presents in childhood but may progress at any age with sight-threatening manifestations (Ranchod et al., 2011). It is characterized by abnormal retinal vascular development with progressive vitreoretinal features, including retinal capillary dropout, vessel dragging; retinal folds, vessel leakage, and exudation; hemorrhage, neovascularization, vitreoretinal interface changes, and serous, tractional, or combined retinal detachment (Wood et al., 2019) – all contributing to or causing visual impairment and blindness. Currently, there are no approved pharmacological therapies for FEVR. For more information, please visit www.caeregen.com.
About Caeregen Therapeutics
Caeregen Therapeutics, LLC, based in Rochester, MI with offices in Chapel Hill, N.C., is a regenerative medicines company developing therapeutics for neurosensory diseases. By exploiting biological pathways and signaling related to cellular and organ development, Caeregen is focused on advancing targeted therapies with the ability to repair, restore and protect neurosensory tissues affected by inherited or acquired diseases. Caeregen is currently developing Noregen™ (CT-107), a unique, novel, recombinant protein mimetic of human norrin-derived growth factor for the potential treatment of retinal-related vision loss. For more information, please visit: www.caeregen.com.
Caeregen Therapeutics, LLC.
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