Gene Therapy for Fanconi Anemia, Complementation Group A

NCTID	NCT04248439 (View at clinicaltrials.gov)
Description	The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure. (Show More)
Development Status	Active
Indication	Fanconi Anemia Complementation Group A
Disease Ontology Term	DOID:0111095
Compound Name	RP-L102
Compound Alias	Mozafancogene autotemcel
Compound Description	PGK-FANCA.WPRE
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	5
Results Posted	Not Available

Target Gene/Variant	FANCA
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	Dose range: 2.0E5 - 4.1E6 transduced CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	MAA accepted by EMA 4/2/24; Rolling BLA submission initiated November 2024