Last update 21 Nov 2024

QR-313

Last update 21 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

ASO

Synonyms

Target

COL7A1

Mechanism

COL7A1 gene modulators

Therapeutic Areas

Immune System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]

Active Indication-

Inactive Indication

Epidermolysis Bullosa DystrophicaEpidermolysis Bullosa Dystrophica, Pasini Type

Originator Organization

ProQR Therapeutics NV

Active Organization-

Inactive Organization

Wings Therapeutics, Inc.Startup

Phoenicis Therapeutics, Inc.

Drug Highest PhaseDiscontinuedPhase 1/2

First Approval Date-

Regulation-

Structure

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Gene Sequence

Sequence Code 1117836319

Clinical Trials associated with QR-313

EUCTR2017-004806-17-ES / Not yet recruitingPhase 1/2IIT

A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS

Start Date06 Sep 2018

Sponsor / Collaborator-

NCT03605069 / TerminatedPhase 1/2

A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With DDEB or RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene

A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis with one or more pathogenic mutations in exon 73 in the COL7A1 gene.

Start Date02 Jul 2018

Sponsor / Collaborator

Phoenicis Therapeutics, Inc.

100 Clinical Results associated with QR-313

100 Translational Medicine associated with QR-313

100 Patents (Medical) associated with QR-313

Literatures (Medical) associated with QR-313

01 Apr 2021·Journal of Investigative DermatologyQ1 · MEDICINE

QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study

Q1 · MEDICINE

Article

Author: Wally, Verena ; Hogervorst, Marieke ; Keene, Douglas R ; Haisma, Elisabeth M ; Hausser, Ingrid ; Nauroy, Pauline ; Nyström, Alexander ; Koller, Ulrich ; Marinkovich, M Peter ; Tufa, Sara F ; Hainzl, Stefan ; Bischof, Johannes ; Swildens, Jim ; Ritsema, Tita ; Platenburg, Gerard ; Murauer, Eva M ; Athanasiou, Ioannis ; Bornert, Olivier ; Kiritsi, Dimitra

Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII. DEB can be inherited as recessive DEB (RDEB) or dominant DEB (DDEB) and is associated with a high wound burden. Perpetual cycles of wounding and healing drive fibrosis in DDEB and RDEB, as well as the formation of a tumor-permissive microenvironment. Prolonging wound-free episodes by improving the quality of wound healing would therefore confer substantial benefit for individuals with DEB. The collagenous domain of collagen VII is encoded by 82 in-frame exons, which makes splice-modulation therapies attractive for DEB. Indeed, antisense oligonucleotide-based exon skipping has shown promise for RDEB. However, the suitability of antisense oligonucleotides for treatment of DDEB remains unexplored. Here, we developed QR-313, a clinically applicable, potent antisense oligonucleotide specifically targeting exon 73. We show the feasibility of topical delivery of QR-313 in a carbomer-composed gel for treatment of wounds to restore collagen VII abundance in human RDEB skin. Our data reveal that QR-313 also shows direct benefit for DDEB caused by exon 73 mutations. Thus, the same topically applied therapeutic could be used to improve the wound healing quality in RDEB and DDEB.

100 Deals associated with QR-313

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Epidermolysis Bullosa Dystrophica, Pasini Type	Phase 2	ES	Phoenicis Therapeutics, Inc.	02 Jul 2018
Epidermolysis Bullosa Dystrophica, Pasini Type	Phase 2	FR	Phoenicis Therapeutics, Inc.	02 Jul 2018
Epidermolysis Bullosa Dystrophica, Pasini Type	Phase 2	US	Phoenicis Therapeutics, Inc.	02 Jul 2018
Epidermolysis Bullosa Dystrophica	Phase 2	US	Wings Therapeutics, Inc.Startup	21 Mar 2018
Epidermolysis Bullosa Dystrophica	Phase 2	FR	Wings Therapeutics, Inc.Startup	21 Mar 2018
Epidermolysis Bullosa Dystrophica	Phase 2	CZ	Wings Therapeutics, Inc.Startup	21 Mar 2018
Epidermolysis Bullosa Dystrophica	Phase 2	GB	Wings Therapeutics, Inc.Startup	21 Mar 2018
Epidermolysis Bullosa Dystrophica	Phase 2	ES	Wings Therapeutics, Inc.Startup	21 Mar 2018
Epidermolysis Bullosa Dystrophica	Phase 2	DE	Wings Therapeutics, Inc.Startup	21 Mar 2018

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