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BioPharm America
BioPharm America is merging with LSX USA
Hynes Convention Center | Boston, MA

September 11–12, 2024

Caeregen Therapeutics

Profile

Caeregen is a breakthrough regenerative medicine therapeutics company targeting neurosensory diseases, with a revolutionary first drug, CTR-107 (Noregen), for vision loss and blindness from retinal diseases. Caeregen’s first-in-class technology generates synthetic human proteins to selectively activate pathways to repair damaged tissues, regenerate new neurovasculature structures and restore normal function. In-vitro and in-vivo studies have shown CTR-107 reactivates the pathways used in initial retinal development to repair and regenerate the blood vessels, capillaries and neuronal structures of the retina to protect and restore vision. Caeregen’s leadership team is comprised of world recognized leaders with >100 years’ experience in retinal disease research, therapeutics, and clinical care, possessing one of the largest independent biobanks of inherited retinal diseases and the largest network of retinal disease clinical research practices in N. America. While CTR-107 has the potential to revolutionize patient care in multi-billion dollar ischemic retinal disease indications such as Retinal Vein Occlusion (RVO), Macular Degeneration (MD), Diabetic Macular Edema (DMA) and Diabetic Retinopathy (DR), Caeregen is first targeting the rare, inherited eye disease of Familial Exudative Vitreoretinopathy (FEVR; no approved treatments), as the fastest, most capital efficient, clinical proof-of-concept and path to market. CTR-107 is in IND-enabling pre-clinical development with its first-in-patient Phase I/II FEVR study to start in 1H24 and initial data in 2H24. The strength of CTR-107’s activity data has enabled robust regulatory progress including US FDA acceptance as a potential therapeutic for FEVR; US FDA and EU EMA attainment Orphan Drug Designation in FEVR; US FDA Rare Pediatric Disease Designation, and; US FDA Priority Review Voucher (PRV) eligibility, (>$100 Million market value at approval).