Release date: 2018-05-18
Recently, Bioverativ and Sangamo Therapeutics jointly announced that the US FDA has accepted the Investigative New Drug (IND) application for the candidate gene therapy BIVV003 for the treatment of sick cell disease. This means that Bioverativ can begin clinical phase 1/2 trials to test the safety, tolerability and efficacy of BIVV003 in patients with sickle cell disease.
Every year, 300,000 people worldwide suffer from sickle cell disease at birth. It is an inherited blood disease caused by a decrease in the ability of hemoglobin to carry oxygen due to a mutation in a gene encoding adult hemoglobin. The patient's red blood cells are sickle-shaped and exhibit symptoms including chronic anemia, vascular occlusion, and multiple organ damage. The patient's risk of stroke increases and life expectancy is shortened.
BIVV003 is an autologous cell therapy developed by Bioverativ and Sangamo using gene editing technology. It collects hematopoietic stem cells (HSCs) from patients and then uses zinc finger nuclease (ZFN) technology to precisely cleave the erythroid enhancer that regulates the BCL11A gene in vitro. This results in a decrease in the expression of the BCL11A gene, which is a transcription factor that inhibits the expression of fetal hemoglobin. An increase in fetal hemoglobin expression can replace the function of adult hemoglobin and alleviate the symptoms of patients with sickle cell disease.
HSCs that have undergone gene editing to enhance fetal hemoglobin expression levels are reinjected into patients, and they can continue to proliferate and differentiate into mature red blood cells. Preclinical trials have shown that genetically engineered red blood cells can increase fetal hemoglobin expression levels by more than four times. The advantage of this therapy is that it does not rely on viral vectors to edit the genes, thus avoiding the possible side effects of viral vectors, and the use of autologous cells can improve the success rate of transplantation and avoid the immunosuppressive process required for allogeneic transplantation.
â–² Dr. Edward Conner, Chief Medical Officer of Sangamo (Source: Sangamo Therapeutics Official Website)
“This is the second IND our gene editing strategy has received from the FDA within a year,†said Dr. Ken Huttner, vice president of clinical development at Bioverativ. “It marks our determination to advance cutting-edge science while waiting for a treatment.†Patients with effective therapies for cytopathic disease have brought hope. We look forward to bringing this project to clinical trials."
"Sickle cell disease is a blood disease that affects a patient's life and causes serious complications. Patients should get more effective new treatment options," said Dr. Edward Conner, Sangamo's chief medical officer. Cell therapy may bring once-and-for-all treatment to patients with sickle cell disease. We believe that the accuracy, efficiency and specificity of ZFN technology make BIVV003 unique."
Bioverativ expects to open several clinical trial sites in the US this year. We look forward to this new therapy to bring the gospel to patients!
Reference materials:
[1] Bioverativ and Sangamo Announce FDA Acceptance of IND Application for Gene-Edited Cell Therapy BIVV003 to TreatSickle Cell Disease
[2] A Potential Therapy for Beta-Thalassemia (ST-400) and Sickle Cell Disease (BIVV003)
Source: WuXi PharmaTech
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