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)
Development Status	Active
Indication	Autosomal Recessive Congenital Ichthyosis
Disease Ontology Term	DOID:0060655
Compound Name	KB105
Sponsor	Krystal Biotech, Inc.
Funder Type	Industry
Recruitment Status	Unknown
Enrollment Count	15
Results Posted	Not Available

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	none
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
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Initiate Phase 2 cohort in 2026