A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

NCTID	NCT04293185 (View at clinicaltrials.gov)
Description	This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD). (Show More)
Development Status	Approved
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	LYFGENIA
Compound Alias	lovotibeglogene autotemcel
Sponsor	Genetix Biotherapeutics Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	35 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	HBB
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	BB305 LV
Editor Type	none
Dose 1	Minimum dose: 3E6 CD34+ cells/kg
Dose 2	Maximum dose: 14E6 CD34+ cells/kg
Dose 3	Median dose: 6.4E6 CD34+ cells/kg
Dose 4
Dose 5

No.of Trial Sites	9
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	FDA approved 12/8/23, price/treatment $3.1M