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MS-R001

Development stage: Phase 1
Lead developer: Santen Pharmaceutical
Modality: Small Molecules
Administration: Ophthalmic

Overview

MS-R001 is an investigational ophthalmic formulation of sirolimus (also known as rapamycin) developed by Santen in collaboration with MacuSight. It is designed for the treatment of diabetic macular edema (DME) secondary to diabetic retinopathy. As an mTOR inhibitor, MS-R001 targets the Mechanistic target of rapamycin kinase, leveraging its immunosuppressive and anti-angiogenic properties to reduce the retinal inflammation and vascular permeability associated with DME. The drug was evaluated in a Phase 1 dose-escalation clinical trial (NCT00401115, also known as DR-001) where it was administered via subconjunctival and intraocular injections to assess safety and tolerability in patients with DME.

Brand names: Opsiria
Other names: sirolimusrapamycin

Targets

FKBP1AmTOR (Mammalian target of rapamycin kinase)

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