Drug intelligence / Profile preview

PF-04523655 + ranibizumab

Development stage
Unknown
Lead developer
Pfizer
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
Administration
Intravitreal
01

Overview

PF-04523655 + ranibizumab is a combination therapy consisting of PF-04523655, a synthetic and chemically modified 19-nucleotide double-stranded small interfering RNA (siRNA) targeting the RTP801 gene, and ranibizumab, a recombinant humanized monoclonal antibody fragment that inhibits vascular endothelial growth factor A (VEGF-A). The combination is administered via intravitreal injection for ophthalmic indications. PF-04523655 acts by silencing RTP801 expression to reduce pathological angiogenesis and vascular leakage in retinal diseases. Ranibizumab binds to VEGF-A, inhibiting its activity and thereby reducing neovascularization and vascular permeability. This combination has been investigated primarily for neovascular age-related macular degeneration (AMD) and diabetic macular edema (DME), with studies suggesting potential additive or synergistic effects on visual acuity improvement compared to monotherapy[1][2][4].

02

Targets

VEGFA (Vascular endothelial growth factor A)DDIT4 (Regulated in development and DNA damage response protein 1)

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