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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency


NCTID NCT06049082 (View at clinicaltrials.gov)
Description
Indication Alpha 1-Antitrypsin Deficiency
Compound Name KB-408
Sponsor Krystal Biotech, Inc.
Funder Type Industry
Status
Recruiting
Enrollment Count 12

Therapy Information


Target Gene/Variant SERPINA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Inhalational
Drug Product Type Viral vector
Target Tissue/Cell Lung
Delivery System Viral transduction
Vector Type HSV-1
Editor Type
Dose 1 10^9 PFU
Dose 2 10^10 PFU
Dose 3 10^11 PFU
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2023-09-15
Completion Date 2026-05
Last Update 2024-05-06

Participation Criteria


Eligible Age 18 Years - 70 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
Recent Updates Phase I Interim data expected 2H 2024

Resources/Links