Current Studies
BEACON
Title of Protocol:
A Phase ½ Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises
Purpose/Objective of Study:
The purpose of the study is to evaluate the safety and ability of the autologous stem cell transplant with gene-edited stem cells to treat your sickle cell disease.
Study Product Description:
BEAM-101 is the investigational product for this clinical trial. BEAM-101 may help treat sickle cell disease by acting to edit or change a person’s genes on purpose to fix an abnormal gene.
Toxicities/Side Effects:
The most frequent side effects are low platelets and mouth sores.
Time Committment: 2.5 years
HGB210
LentiGlobin for Sickle Cell Disease Gene Therapy Overview
DP, drug product; HSCs, hematopoietic stem cells; RBCs, red blood cells
Title of Protocol:
A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease
Purpose/Objective of Study:
The primary purpose of this study is to evaluate the efficacy and safety of treatment with LentiGlobin BB305 Drug Product and autologous transplant in individuals with severe sickle cell disease (SCD).
Study Agent(s) and Mechanism of Drug Action/Device Description:
LentiGlobin BB305 Lentiviral Vector: LentiGlobin BB305 LVV is a replication defective, self-inactivating HIV-1 based vector pseudotyped with the vesicular stomatitis virus G envelope protein, carrying the human β-globin gene with a single modification in the coding region at codon 87 (βA87 Thr:Gln [βA-T87Q]).
LentiGlobin BB305 Drug Product: LentiGlobin BB305 Drug Product is defined as an autologous CD34+ cell-enriched population that contains HSCs transduced with LentiGlobin BB305 LVV encoding the βA-T87Q-globin gene, suspended in cryopreservation solution in the final immediate container for the intended medical use.
Toxicities/Side Effects:
The most frequent adverse events thrombocytopenia and mucositis.
Time Committment: 2.5 years
LTF-307
Title of Protocol:
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
Purpose/Objective of Study:
The primary purpose of this study is to evaluate the long-term safety and efficacy of the gene therapy drug product used in bluebird bio-sponsored clinical studies in treated subjects with sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
BB305 Lentiviral Vector: LentiGlobin BB305 LVV is a replication defective, self-inactivating HIV-1 based vector pseudotyped with the vesicular stomatitis virus G envelope protein, carrying the human β-globin gene with a single modification in the coding region at codon 87 (βA87 Thr:Gln [βA-T87Q]).
BB305 Drug Product: LentiGlobin BB305 Drug Product is defined as an autologous CD34+ cell-enriched population that contains HSCs transduced with LentiGlobin BB305 LVV encoding the βA-T87Q-globin gene, suspended in cryopreservation solution in the final immediate container for the intended medical use.
Toxicities/Side Effects:
There is no treatment intervention for this study- long term follow up only.
Time Committment: 13 years