AAV2-hAADC

Phase 1Gene therapy

KKrzysztof Bankiewicz

AAV2-hAADC is one of 4 small molecule programs targeting AADC Deficiency. It is currently in Phase 1.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

AADC DeficiencyRecruiting

Parkinson's Disease (PD)Not yet recruiting

AADC DeficiencyPhase 1

Recruiting

Parkinson's Disease (PD)Phase 1

Not yet recruiting

Competition

Competitive Landscape

8 competitors in Parkinson's Disease (PD)

#DrugCompanyPhaseTrials

Glucophage

EMD Serono

Approved

1 2

TJ0113

HHangzhou PhecdaMed Co., Ltd.

Phase 3

1 3

AAV2-hAADC

KKrzysztof Bankiewicz

Phase 1

1 4

Buntanetap/posiphen

AAnnovis Bio Inc.

Preclinical

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

Timeline

Expected Milestones

Q4 2028

Phase 1Not yet recruiting

Parkinson's Disease (PD)

NCT07267065

Q3 2037

Phase 1Recruiting

AADC Deficiency

NCT02852213

Clinical

Trials by Indication[1]

Clinical Trial1 trial

A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion

Completed·NNational Taiwan University Hospital·Phase 2·NCT02926066·12 enrolled

AADC Deficiency1 trial

SIngle-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency

Recruiting·KKrzysztof Bankiewicz·Phase 1·NCT02852213·42 enrolled

Parkinson's Disease (PD)1 trial

An Open-label Safety and Efficacy Study of AAV2-hAADC Administered by MRI-Guided Convective Infusion Using a Transfrontal Trajectory Into the Putamen and Caudate of Participants With Young Onset Parkinson's Disease

Not yet recruiting·KKrzysztof Bankiewicz·Phase 1·NCT07267065·9 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for AAV2-hAADC. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — AAV2-hAADC compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for AAV2-hAADC. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Krzysztof Bankiewicz company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Gene therapy[2]
Highest Phase: Phase 1[1]
Company: KKrzysztof Bankiewicz

AAV2-hAADC

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2028

Q3 2037

Trials by Indication[1]

Clinical Trial1 trial

AADC Deficiency1 trial

Parkinson's Disease (PD)1 trial

References

Drug Info

Quick Facts

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