Caeregen Therapeutics has received a Phase 2 Small Business Innovation Research (SBIR) grant worth $1.4 million from the National Institutes of Health (NIH) and National Eye Institute (NEI).

The grant is intended to support the further development of CTR-107 (Noregen), a novel regenerative therapeutic designed to address vision loss associated with various inherited and age-related retinal diseases.

The initial focus of Caeregen’s development efforts for CTR-107 is on the treatment of Familial Exudative Vitreoretinopathy (FEVR), a rare inherited disorder characterised by abnormal blood vessel formation in the retina.

With the support of the SBIR grant, Caeregen Therapeutics aims to advance the clinical development of CTR-107, to provide an innovative treatment option for individuals experiencing retinal-related vision loss caused by a broad range of inherited and age-related retinal diseases.

Caeregen Therapeutics CEO Walter Capone said: “The NIH grant will support the nonclinical development program of CTR-107 and accelerate our investigational new drug (IND) submission to begin clinical trials.”

Caeregen Therapeutics chief scientific officer Kimberly Drenser said: “This award provides further, important validation for CTR-107 and our novel approach to retinal disease treatment, the first with the potential to restore retinal function and reverse vision loss.”

Retinal diseases, including macular degeneration, diabetic retinopathy, retinal vein occlusion, and ischemia, are the leading cause of vision loss in developed countries and the fastest-growing cause of blindness globally. These conditions affect millions of people worldwide. In addition to age-related and acquired retinal disorders, there are also rare genetic conditions such as Norrie Disease, Osteoporosis Pseudoglioma Syndrome, and Familial Exudative Vitreoretinopathy (FEVR) that result in significant vision impairment.

Current treatments for retinal diseases can slow down the progression of vision loss but are unable to repair or reverse existing retinal damage. There is a need for therapies that can restore retinal function and counteract vision loss.

CTR-107, developed by Caeregen Therapeutics, is a synthetic targeted growth factor that mimics the function of norrin, a naturally occurring human protein. Norrin promotes the development of organized blood vessels and neurons in the eye, ear, and central nervous system. By injecting CTR-107 into the eye, it is believed that the therapy can regenerate retinal blood vessels and neurons, thereby restoring retinal function and addressing vision loss associated with conditions like FEVR and other retinal diseases.

The Phase 2 Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) and National Eye Institute (NEI) provides support for the further development of CTR-107. The grant will allow Caeregen to optimise dosing through in vitro and in vivo studies, refine analytical methods for product characterisation, and conduct pharmacokinetic studies to support an IND (Investigational New Drug) submission.

CTR-107 has obtained Rare Pediatric Disease (RPD) Designation from the US FDA for the treatment of FEVR, making Caeregen eligible to receive a Priority Review Voucher upon marketing approval.

Additionally, both the FDA and the European Commission European Medicines Agency (EMA) have granted Orphan Drug Designations to CTR-107. These designations recognise the therapeutic potential of CTR-107 for rare diseases and provide certain benefits and incentives to support its development.