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QR-313
- Development stage
- Phase 2
- Lead developer
- Phoenicis Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Topical
01
Overview
QR-313 is a first-in-class, RNA-based antisense oligonucleotide designed for the treatment of dystrophic epidermolysis bullosa (DEB), specifically targeting mutations in exon 73 of the COL7A1 gene. DEB is a severe genetic skin disorder characterized by blistering and poor wound healing due to loss of functional collagen type VII protein, which is essential for anchoring fibrils that connect the dermal and epidermal layers of the skin. QR-313 works by inducing exon skipping during mRNA splicing, thereby excluding exon 73 from COL7A1 mRNA and enabling production of a functional collagen VII protein. This approach aims to restore anchoring fibril function and improve wound healing in patients with both recessive (RDEB) and dominant (DDEB) forms caused by relevant mutations. The drug is formulated as a topical gel for direct application to wounds[1][2][4][5][7].
- Other names
- PTW-002PTW002PTW 002
02
Targets
COL7A1 (Collagen type VII alpha 1 chain)
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