BD211 / BDgene - LARVOL DELTA

Preclinical efficacy and safety evaluation of BD211 autologous CD34+ hematopoietic stem cell injection for transfusion-dependent β-thalassemia in NCG-X mice. (PubMed, Front Cell Dev Biol) - "The no observed adverse effect level of BD211 was established at 1.2 × 106 cells per mouse. BD211 shows potential as a safe therapeutic approach for treating transfusion-dependent thalassemia."

Journal • Preclinical • Beta-Thalassemia • Genetic Disorders • CD34

A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants (clinicaltrials.gov) - P=N/A | N=2 | Completed | Sponsor: Shanghai BDgene Co., Ltd. | N=10 ➔ 2 | Trial primary completion date: Feb 2025 ➔ Aug 2024 | Recruiting ➔ Completed

Enrollment change • Trial completion • Trial primary completion date • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Transplantation

A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Shanghai BDgene Co., Ltd.

New P1 trial • Beta-Thalassemia • Genetic Disorders

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia (clinicaltrials.gov) - P=N/A | N=10 | Recruiting | Sponsor: Shanghai BDgene Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Viral vector • Beta-Thalassemia • Genetic Disorders

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia (clinicaltrials.gov) - P=N/A | N=10 | Recruiting | Sponsor: Shanghai BDgene Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Viral vector • Beta-Thalassemia • Genetic Disorders

Modified lentiviral globin gene therapy for pediatric β0/β0 transfusion-dependent β-thalassemia: A single-center, single-arm pilot trial. (PubMed, Cell Stem Cell) - P=N/A | "Here, we report the interim analysis of a single-center, single-arm pilot trial (NCT05015920) evaluating the safety and efficacy of a β-globin expression-optimized and insulator-engineered lentivirus-modified cell product (BD211) in β0/β0 TDT...Single-cell DNA/RNA-sequencing analysis of the dynamic changes of gene-modified cells, transgene expression, and oncogene activation showed no notable adverse effects. Optimized lentiviral gene therapy may safely and effectively treat all β-thalassemia."

Gene therapy • Journal • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Oncology • Pediatrics

A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants (clinicaltrials.gov) - P=N/A | N=10 | Recruiting | Sponsor: Shanghai BDgene Co., Ltd. | Trial completion date: Feb 2024 ➔ Feb 2025 | Trial primary completion date: Feb 2024 ➔ Feb 2025

Gene therapy • Trial completion date • Trial primary completion date • Viral vector • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Transplantation

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia (clinicaltrials.gov) - P=N/A | N=10 | Not yet recruiting | Sponsor: Shanghai BDgene Co., Ltd.

New trial • Viral vector • Genetic Disorders

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia (clinicaltrials.gov) - P=N/A | N=10 | Not yet recruiting | Sponsor: Shanghai BDgene Co., Ltd.

New trial • Viral vector • Beta-Thalassemia • Genetic Disorders