Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

NCTID	NCT05739643 (View at clinicaltrials.gov)
Description	This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group. (Show More)
Indication	Krabbe Disease
Compound Name	FBX-101
Sponsor	Forge Biologics, Inc
Funder Type	Industry
Status	Recruiting
Enrollment Count	9

Target Gene/Variant	GALC
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh10
Editor Type
Dose 1	1.6 x 10^13 gc/kg
Dose 2	Undisclosed dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
Recent Updates	ASGCT 2024: Update given on Phase 1b data; dose escalation to highest dose approved, will start 2Q24