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PF-04523655
- Development stage
- Phase 2
- Lead developer
- Quark Pharmaceuticals
- 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
- Administration
- Intravitreal
01
Overview
PF-04523655 is a chemically stabilized small interfering RNA (siRNA) therapeutic targeting the RTP801 gene (also known as REDD1). RTP801 is an inhibitor of the mammalian target of rapamycin complex 1 (mTORC1) and downstream transcription factor HIF-1. By silencing RTP801 via RNA interference, PF-04523655 aims to modulate cellular stress responses and promote cell survival in retinal diseases. The drug was developed for intravitreal administration to treat eye disorders such as diabetic macular edema (DME) and neovascular (wet) age-related macular degeneration (AMD). Clinical studies have shown that it can improve visual acuity in DME patients with a favorable safety profile[4][5][6][7][8].
- Other names
- RTP801I-14RTP-801I-14RTP 801I-14RTP801i14RTP-801i14RTP 801i14
02
Targets
DDIT4 (Regulated in development and DNA damage response protein 1)
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