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QPI-1007
- Development stage
- Phase 2
- Lead developer
- Quark Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravitreal
01
Overview
QPI-1007 is a synthetic, double-stranded, chemically modified small interfering RNA (siRNA) developed by Quark Pharmaceuticals. It is designed to temporarily inhibit the expression of caspase 2, a pro-apoptotic protein involved in the apoptotic death of retinal ganglion cells. By blocking caspase 2 expression through RNA interference, QPI-1007 acts as a neuroprotectant for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION) and potentially other optic neuropathies such as glaucoma. The drug is administered via intravitreal injection and has demonstrated safety and some efficacy in early-phase clinical trials. QPI-1007 has received orphan drug designation from the US FDA for optic nerve disorders.
- Other names
- cosdosiran
02
Targets
Caspase-2 messenger RNA (CASP2 mRNA)
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