AAV2-hRPE65v2,voretigene neparvovec-rzyl

Phase 3Gene therapy

AAV2-hRPE65v2,voretigene neparvovec-rzyl is one of 5 biologic programs targeting Leber Congenital Amaurosis. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Leber Congenital AmaurosisActive, not recruiting

Leber Congenital AmaurosisPhase 3

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

Q1 2030

Phase 3Active, not recruiting

Leber Congenital Amaurosis

NCT00999609

Clinical

Trials by Indication[1]

Leber Congenital Amaurosis1 trial

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Active, not recruiting·SSpark Therapeutics, Inc.·Phase 3·NCT00999609·31 enrolled

GIST1 trial

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Completed·SSpark Therapeutics, Inc.··NCT03597399·87 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for AAV2-hRPE65v2,voretigene neparvovec-rzyl. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — AAV2-hRPE65v2,voretigene neparvovec-rzyl compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for AAV2-hRPE65v2,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 3[1]
Company: SSpark Therapeutics, Inc.

AAV2-hRPE65v2,voretigene neparvovec-rzyl

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2030

Trials by Indication[1]

Leber Congenital Amaurosis1 trial

GIST1 trial

References

Drug Info

Quick Facts

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