Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

NCTID	NCT05735158 (View at clinicaltrials.gov)
Description	KB105-02 is an intrasubject randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of KB105 in children and adults with autosomal recessive congenital ichthyosis (ARCI). (Show More)
Indication	Autosomal Recessive Congenital Ichthyoses, TGM1-deficient
Compound Name	KB-105
Sponsor	Krystal Biotech, Inc.
Funder Type	Industry
Status	Not yet recruiting
Enrollment Count	15

Target Gene/Variant	TGM1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Topical
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	HSV-1
Editor Type
Dose 1	Undisclosed single dose formulated as a topical gel, weekly application
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
Recent Updates	Initiate Phase 2 cohort in 2024