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PNHB + liposomal sirolimus is a combination formulation consisting of poly(N-hexyl-butyric acid) (PNHB) and sirolimus encapsulated in a liposomal delivery system. Liposomal sirolimus is an investigational, controlled-release, nanoparticle drug formulation designed to optimize sirolimus pharmacokinetics for local delivery, notably in ocular and vascular tissues. Sirolimus is a macrocyclic lactone compound functioning as an mTOR (mechanistic target of rapamycin) inhibitor that acts as an immunosuppressant, antiproliferative, and anti-inflammatory agent. The liposomal encapsulation aims to enhance local tissue distribution, reduce systemic exposure, and enable sustained release of sirolimus, potentially improving safety and efficacy in conditions such as autoimmune ophthalmopathies, transplant-related organ rejection, and vascular restenosis. PNHB may be a carrier or stabilizing polymer in the formulation (information on PNHB alone is limited in current sources). Development has focused on local delivery (e.g., subconjunctival injection, vascular wall application) to target tissues while minimizing off-target effects[1][2][3].
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