Caeregen Therapeutics Enhances Retinal Health with $1.4 Million NIH/NEI Grant: Noregen™ (CTR-107) to Tackle Retinal Diseases

Caeregen Therapeutics today announced that it has received $1.4 million from the NIH and NEI in the form of a Small Business Innovation Research (SBIR) grant to develop CTR-107 (Noregen™), a novel regenerative therapeutic designed to treat retinal-related vision loss caused by an array of inherited or age-related diseases. The initial focus of the project is directed at Familial Exudative Vitreoretinopathy (FEVR), a rare hereditary disorder of retinal blood vessel development.

Caeregen Therapeutics is thrilled to announce that they have been awarded an NIH grant to further the nonclinical development of their investigational drug, CTR-107. This grant allows the company to rapidly advance to their IND submission and the start of clinical trials.

CTR-107 is the first treatment with the potential to restore retinal function and reverse vision loss, making the NIH grant a formidable validation for Caeregen’s novel approach to retinal disease treatment.

CEO Walter Capone and Chief Scientific Officer Dr. Kimberly Drenser are both encouraged by this development and believe it will re-energize the medical field’s prospects for vision restoration.

Retinal diseases are an ominous public health problem that is slowly taking a firm grip on modern society. Affecting more than 10 million people across the United States and Europe alone, and over 50 million people worldwide, sufferers are dealing with vision deprivation in the forms of macular degeneration, diabetic retinopathy, retinal vein occlusion and ischemia.

Even rare genetic retinal diseases like Norrie Disease, Osteoporosis Pseudoglioma Syndrome and FEVR can cause devastating vision loss. Retinal disease has become the leading cause of vision loss in developed countries and is said to be the fastest growing cause of global blindness.

Retinal disorders, whether acquired or age-related, have a common denominator: an unfortunate deterioration of the retinal vascular bed and associated neurovascular structure. Fortunately, existing treatments can help delay further vision loss, however they cannot fix existing damage to the retina.

CTR-107, a synthetic targeted growth factor that mimics the properties of the natural human protein norrin, could be a breakthrough in restoring sight to those suffering from degenerative eye diseases.

By helping to stimulate the development of organized blood vessels and neurons in the eye, ear, and CNS, CTR-107 holds the promise of restoring retinal function and reversing vision loss associated with FEVR and other retinal diseases when injected into the eye.

Building on the success of our Phase 1 STTR grant, including production feasibility, safety/tolerability, target gene expression modulation, and in vivo functional activity in vitro, we are now advancing to Phase 2 of the SBIR grant.

During this phase, we will be optimizing the dosing of our product through in vitro and in vivo studies. We will also continue to develop analytical methods to accurately characterize our product and conduct pharmacokinetic studies to provide the crucial foundation of an IND submission. Ultimately, this research will allow us to make a greater impact with our innovative product.

Caeregen’s CTR-107 (Noregen™) has made history by becoming the first program to obtain U.S. FDA’s Rare Pediatric Disease (RPD) Designation for treatment of FEVR, a designation that could see the program eligible to obtain a Priority Review Voucher should the program gain marketing approval. Adding to the milestone, it has also earned Orphan Drug Designations from both the FDA and European Medicines Agency (EMA).

About CTR-107 (Noregen™)

CTR-107, also known as Noregen™, is a revolutionary and first-of-its-kind synthetic targeted growth-factor modeled after norrin, a naturally occurring human protein. This innovative compound has the potential to promote the development of organized blood vessels and neurons in the eye, ear, and central nervous system of individuals afflicted by inherited or acquired retinal diseases.

Clinical trials are currently underway to explore its application in Familial Exudative Vitreoretinopathy (FEVR), a rare and urgently unmet medical need. With CTR-107, there is newfound hope that retina-related vision loss may be reversed and vision restored–opening up a wealth of possibilities for those whose lives have been touched by this devastating condition.


Familial Exudative Vitreoretinopathy (FEVR) is an inherited disorder of retinal angiogenesis, leading to incomplete vascularization of the peripheral retinal and poor vascular differentiation. Unsurprisingly, this rare condition is most prevalent in children, but can continue to progress at any age, with even the potential for sight-threatening manifestations.

These manifestations include retinal capillary dropout, retinal folds, vessel leakage, exudation, hemorrhage, neovascularization, vitreoretinal interface changes, and various forms of retinal detachment – all of which carry the risk of visual impairment and/or blindness. Unfortunately, despite the prevalence of this condition, there are currently no approved pharmacological treatments available to address FEVR.

About Caeregen Therapeutics

Caeregen Therapeutics, LLC, a regenerative medicines company with offices in both Rochester, MI and Chapel Hill, NC, is working diligently to develop innovative therapeutics for the treatment of neurosensory diseases.

Taking a unique approach by focusing on pathways and signaling related to tissue and organ development, Caeregen seeks to bring to fruition targeted therapies that help to repair, restore, and protect neurosensory tissues that have been damaged or affected by inherited or acquired diseases.

One of Caeregen’s most ambitious endeavors is Noregen, a powerful recombinant protein with the ability to mimic the effects of human norrin-derived growth factor that could potentially revolutionize the way vision loss due to retinal issues is treated.

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