sepofarsen

Phase 3RNA therapy

PProQR Therapeutics

sepofarsen is one of 53 small molecule programs targeting Efficacy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

EfficacyActive, not recruiting

Leber Congenital AmaurosisRecruiting

EfficacyPhase 3

Active, not recruiting

Leber Congenital AmaurosisPhase 3

Recruiting

Competition

Competitive Landscape

139 competitors in Efficacy

#DrugCompanyPhaseTrials

Rayos

RRoxane Laboratories

Phase 3

5 2

Cytoxan

BBaxter Healthcare Corporation

Phase 3

3 3

Alimta

SShawn Eagle

Phase 3

3 4

Bystolic

VVitae Pharmaceuticals Inc., an Allergan affiliate

Phase 3

2 5

Pifeltro

CCidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Phase 3

1 6

sepofarsen

PProQR Therapeutics

Phase 3

1 7

Veracept

SSebela Women's Health Inc.

Phase 3

1 8

Levocept

SSebela Women's Health Inc.

Phase 3

1 9

Aralen

Sanofi

Preclinical

2 10

Primaquine

Sanofi

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2028

Phase 3Recruiting

Leber Congenital Amaurosis

NCT06891443

Clinical

Trials by Indication[1]

Efficacy1 trial

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Active, not recruiting·PProQR Therapeutics·Phase 2/3·NCT03913143·36 enrolled

Randomized1 trial

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

Unknown status·PProQR Therapeutics·Phase 2/3·NCT04855045·15 enrolled

Leber Congenital Amaurosis1 trial

A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene

Recruiting·LLaboratoires Thea·Phase 3·NCT06891443·32 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for sepofarsen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — sepofarsen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for sepofarsen. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — ProQR Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: RNA therapy[2]
Highest Phase: Phase 3[1]
Company: PProQR Therapeutics

sepofarsen

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2028

Trials by Indication[1]

Efficacy1 trial

Randomized1 trial

Leber Congenital Amaurosis1 trial

References

Drug Info

Quick Facts

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