The National Institutes of Health (NIH) and National Eye Institute (NEI) have awarded Caeregen Therapeutics, LLC. a $1.4 million Phase 2 Small Business Innovation Research (SBIR) grant to support the development of CTR-107, the company’s regenerative medicine clinical candidate for retinal diseases.
Let’s start with this company.
Based in Rochester, Michigan, Caeregen is a regenerative medicine company focused on developing targeted therapies that repair, restore, and protect neurosensory tissues affected by inherited or acquired diseases such as:
- Familial exudative vitreoretinopathy (FEVR)
- Norrie Disease
- Osteoporosis-pseudoglioma syndrome (OPPG)
Now CTR-107.
CTR-107 (Noregen) is a novel regenerative therapeutic that is initially being studied for the treatment of FEVR, a rare inherited genetically heterogeneous disorder associated with abnormal retinal blood vessel formation (angiogenesis).
Of note, a “genetically heterogeneous” condition is one that could be inherited in an autosomal dominant, autosomal recessive, or X-linked recessive manner.
Go on …
The synthetic targeted growth-factor is based on norrin—a naturally-occurring human protein that guides retinal formation in fetal development.
Further, the therapeutic is also being explored for treatment of retinal vein occlusion (RVO), in which a phase 1a/b dose escalation study is planned.
What is it designed to do?
CTR-107 is designed to promote the development of organized blood vessels and neurons in the human eye, ear, and central nervous system, with the potential to regenerate and repair the retinal vasculature.
The goal: to reverse vision loss in diseases affecting retinal and choroidal blood vessels.
What else to know?
The CTR-107 program is the first to receive a FDA Rare Pediatric Disease (RPD) designation—which is granted to sponsors of serious or life-threatening diseases that affect less than 200,000 people in the United States—for FEVR as well as Orphan Drug designation (in January 2022).
With this designation, the company is eligible to obtain a priority review voucher when seeking marketing approval.
Gotcha. Now talk about this grant.
The Phase 2 SBIR grant is part of the SBIR and Small Business Technology Transfer (STTR) programs that encourage U.S.-based small businesses to engage in federal research / research and development with the potential for commercialization via collaboration with a research institution.
The SBIR grant intended to provide funding for a small business to continue research and development of a specific technology.
Subsequently, a company may only submit one application for this grant; if rejected, the company is unable to resubmit.
How will it support CTR-107?
According to Caeregen’s CEO Walter Capone, it will be used to “support the nonclinical development program of CTR-107 and accelerate our investigational new drug (IND) submission to begin clinical trials.”
Give me more.
The company is planning to use the grant-funded research to:
- Optimize dosing through in vitro and in vivo studies
- Develop further analytical methods for product characterization
- Conduct pharmacokinetic studies (to determine how the drug may affect the human body) in support of a potential IND submission
Significance?
Per the company, CTR-107 has the potential to be the first regenerative therapy for the treatment of FEVR-associated vision loss.
So stay tuned for clinical updates in the near future!