Explore a selection of our essential drug information below, or:
Identification
- Generic Name
- RTP-801i
- DrugBank Accession Number
- DB06048
- Background
RTP-801i is an siRNA drug candidate designed to inhibit the expression of the hypoxia-inducible gene RTP801. The proposed primary indication for RTP-801i is the treatment of patients with neovascular (wet) Age-related Macular Degeneration (AMD or ARMD).
- Type
- Biotech
- Groups
- Investigational
- Biologic Classification
- Protein Based Therapies
Other protein based therapies - Protein Chemical Formula
- Not Available
- Protein Average Weight
- Not Available
- Sequences
- Not Available
- Synonyms
- Not Available
- External IDs
- PF 04523655
- PF-04523655
- PF-655
- PFE-4523655
- REDD-14-NP
Pharmacology
- Indication
Investigated for use/treatment in macular degeneration.
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Not Available
- Mechanism of action
RTP-801i is an siRNA drug candidate designed to inhibit the expression of the hypoxia-inducible gene RTP-801. The gene RTP-801 was discovered by Quark and initially identified by its dramatic up-regulation in response to hypoxia and/or oxidative stress both in vitro and in animal models. Since expression of RTP-801 is rapidly upregulated in response to ischemia, hypoxia and/or oxidative stress, it represents a unique gene target that may regulate hypoxia-induced pathogenesis by a mechanism that is independent of growth factors such as VEGF. This hypoxia-inducible gene promotes neuronal cell apoptosis and the generation of reactive oxygen species in vitro. In both genetic (RTP801-knockout) and therapeutic mouse and primate models of laser-induced choroidal neovascularization (CNV), inhibition of RTP801 expression leads to inhibition or reduction of CNV and vessel leakage following intravitreal injection of RTP-801i.
Target Actions Organism UDNA damage-inducible transcript 4 protein Not Available Humans - Absorption
Not Available
- Volume of distribution
Not Available
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
Not Available
- Clearance
Not Available
- Adverse Effects
Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates. View sample adverse effects data in our new Data Library!Improve decision support & research outcomes with our structured adverse effects data.- Toxicity
Not Available
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- Not Available
Categories
- Drug Categories
- Chemical TaxonomyProvided by Classyfire
- Description
- Not Available
- Kingdom
- Organic Compounds
- Super Class
- Organic Acids
- Class
- Carboxylic Acids and Derivatives
- Sub Class
- Amino Acids, Peptides, and Analogues
- Direct Parent
- Peptides
- Alternative Parents
- Not Available
- Substituents
- Not Available
- Molecular Framework
- Not Available
- External Descriptors
- Not Available
- Affected organisms
- Not Available
Chemical Identifiers
- UNII
- C9G13J4D49
- CAS number
- Not Available
References
- General References
- PR Newswire: In a Phase 2 Study PF-04523655 (RTP801I-14) Showed Improved Vision Over Standard of Care in Patients with Diabetic Macular Edema at 12 Months [Link]
- External Links
- Not Available
Clinical Trials
- Clinical Trials
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample data2 Completed Treatment Age - Related Macular Degeneration (AMD) 1 somestatus stop reason just information to hide 2 Completed Treatment Diabetic Macular Edema (DME) / Diabetic Retinopathy (DR) / Subfoveal Choroidal Neovascularization (CNV) 1 somestatus stop reason just information to hide 2 Terminated Treatment Diabetes Complications / Diabetic Retinopathy (DR) 1 somestatus stop reason just information to hide 1 Completed Treatment Age - Related Macular Degeneration (AMD) 1 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
- Not Available
- Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Solid
- Experimental Properties
- Not Available
Targets
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- Unknown
- General Function
- Regulates cell growth, proliferation and survival via inhibition of the activity of the mammalian target of rapamycin complex 1 (mTORC1). Inhibition of mTORC1 is mediated by a pathway that involves DDIT4/REDD1, AKT1, the TSC1-TSC2 complex and the GTPase RHEB. Plays an important role in responses to cellular energy levels and cellular stress, including responses to hypoxia and DNA damage. Regulates p53/TP53-mediated apoptosis in response to DNA damage via its effect on mTORC1 activity. Its role in the response to hypoxia depends on the cell type; it mediates mTORC1 inhibition in fibroblasts and thymocytes, but not in hepatocytes (By similarity). Required for mTORC1-mediated defense against viral protein synthesis and virus replication (By similarity). Inhibits neuronal differentiation and neurite outgrowth mediated by NGF via its effect on mTORC1 activity. Required for normal neuron migration during embryonic brain development. Plays a role in neuronal cell death
- Specific Function
- 14-3-3 protein binding
- Gene Name
- DDIT4
- Uniprot ID
- Q9NX09
- Uniprot Name
- DNA damage-inducible transcript 4 protein
- Molecular Weight
- 25370.225 Da
Drug created at November 18, 2007 18:29 / Updated at July 26, 2024 04:51