voretigene neparvovec-rzyl

Phase 2Gene therapy

voretigene neparvovec-rzyl is one of 5 biologic programs targeting Leber Congenital Amaurosis. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Leber Congenital AmaurosisActive, not recruiting

Leber Congenital AmaurosisPhase 2

Active, not recruiting

Competition

Competitive Landscape

8 competitors in Leber Congenital Amaurosis

#DrugCompanyPhaseTrials

Sepofarsen

PProQR Therapeutics

Phase 3

1 2

AAV2-Hrpe65v2,voretigene Neparvovec-Rzyl

SSpark Therapeutics, Inc.

Phase 3

1 3

Rayos

RRoxane Laboratories

Preclinical

1 4

voretigene neparvovec-rzyl

SSpark Therapeutics, Inc.

Phase 2

2 5

Triamcinalone Acetonide

AAtsena Therapeutics Inc.

Preclinical

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

Timeline

Expected Milestones

Q4 2027

Phase 1/2Active, not recruiting

Leber Congenital Amaurosis

NCT01208389

Clinical

Trials by Indication[1]

Leber Congenital Amaurosis2 trials

Safety Study in Subjects With Leber Congenital Amaurosis

Completed·SSpark Therapeutics, Inc.·Phase 1·NCT00516477·12 enrolled

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Active, not recruiting·SSpark Therapeutics, Inc.·Phase 1/2·NCT01208389·12 enrolled

Sources

References

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

Details

Drug Info

Quick Facts

Modality: Gene therapy[2]
Highest Phase: Phase 2[1]
Company: SSpark Therapeutics, Inc.

voretigene neparvovec-rzyl

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Trials by Indication[1]

Leber Congenital Amaurosis2 trials

References

Drug Info

Quick Facts

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