OF-01, a topical dual-chaperone therapy for trabecular meshwork restoration.
A fixed-dose combination of two clinically characterized chemical chaperones, formulated as a single topical eye drop, designed to address the cellular pathology that drives outflow failure in glaucoma.
A dual-chaperone rationale for outflow restoration.
Glaucomatous trabecular meshwork shows elevated endoplasmic reticulum stress, impaired protein handling, mitochondrial dysfunction, and progressive fibrosis. OF-01 pairs two agents that act on those axes.
PBA · proteostasis
Sodium 4-phenylbutyrate acts as a chemical chaperone, intended to support protein folding and reduce endoplasmic reticulum stress and the unfolded-protein-response burden that accumulates in the glaucomatous meshwork.
TUDCA · membrane stability
Tauroursodeoxycholic acid is intended to stabilize ER and mitochondrial membranes and support cellular quality control, reducing apoptosis, inflammation and oxidative injury in stressed meshwork cells.
Reduced fibrosis
Design goal: lower fibrotic signaling and extracellular matrix accumulation in the trabecular meshwork, addressing the structural stiffening that raises conventional outflow resistance.
Improved outflow
Intended result: increased conventional outflow facility and reduced intraocular pressure, acting on the tissue that controls drainage rather than suppressing aqueous production.
OF-01 is investigational and has not been shown to be safe or effective in humans.
Investigational. Product image is a representation of the intended presentation; final formulation, concentrations and container-closure system are being established during IND-enabling work.
A once-daily eye drop, in a standard bottle.
A sterile topical ophthalmic solution, fixed-dose 4-PBA + TUDCA, delivered from a conventional multi-dose dropper. No device, no procedure, no change to the daily routine.
sodium 4-phenylbutyrate (PBA)
Clinically characterized in urea-cycle disorders with an established systemic safety database.
tauroursodeoxycholic acid (TUDCA)
Established safety profile with demonstrated neuroprotective and cellular stress activity.