AAV2-hRPE65v2,voretigene neparvovec-rzyl

Phase 3Gene therapy

AAV2-hRPE65v2,voretigene neparvovec-rzyl is one of 3 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

7 competitors in Leber Congenital Amaurosis

#DrugCompanyPhaseTrials

AAV2-hRPE65v2,voretigene neparvovec-rzyl

SSpark Therapeutics, Inc.

Phase 3

1 2

Rayos

RRoxane Laboratories

Preclinical

1 3

Voretigene Neparvovec-Rzyl

SSpark Therapeutics, Inc.

Phase 2

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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