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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
NCTID
NCT05504837
(View at clinicaltrials.gov)
Description
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
(Show More)
Indication
Cystic Fibrosis
Compound Name
KB-407
Sponsor
Krystal Biotech, Inc.
Funder Type
Industry
Status
Recruiting
Enrollment Count
12
Therapy Information
Target Gene/Variant
CFTR
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
Delivery System
Viral transduction
Vector Type
HSV-1
Editor Type
Dose 1
Undisclosed dose (single administration)
Dose 2
Undisclosed dose (2 administrations)
Dose 3
Undisclosed dose (4 administrations)
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2022-08-15
Completion Date
2024-07
Last Update
2024-04-09
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
2
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
Completed dosing in first two cohorts and on track to start third in Q2 2024
Resources/Links
Patents
WO2020163703A1
WO2021127524A1
Clinical Publications
https://ir.krystalbio.com/static-files/8b395c9d-3d0a-4e8e-be20-023ff9c475bb
News and Press Releases
https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-first-quarter-2024-financial-results
Preclinical Publications
https://ir.krystalbio.com/static-files/4daf5858-70fc-4193-93d4-6ccb85fb53ef
https://ir.krystalbio.com/static-files/04c0187d-1f2f-439d-935e-20980ed6729d
https://ir.krystalbio.com/static-files/efd3ab11-ea4a-4bf0-a5d5-66db0e8bc4e5