However, results from this small, single-arm pilot study will need to be validated in larger, longer-term studies. At the latest follow-up (which ranged from 1 to 18 months post–gene therapy), total HbF levels had increased to between 23.8% and 42.8%, and remained stable, Dr. Esrick said. Knocking down BCL11A using a lentiviral vector-based approach resulted in effective induction of fetal hemoglobin and significant attenuation of the sickling phenotype, with no vector-related adverse events, investigator Erica B. Esrick, MD, of Children’s Hospital Boston, said during the meeting’s late-breaking abstracts session.
A BCL11A-targeting gene therapy in patients with sickle cell disease (SCD) led to higher levels of fetal hemoglobin (HbF) and reductions in the severity of disease, according to findings from a small pilot study.
Use of this Web site is subject to the medical disclaimer. “At the 3-month time point before re-starting transfusions, the subject with moyamoya had a pre-transfusion Hb of 11 g/dL with 76% of non-transfused cells containing on average 17pg F/F cell,” the authors wrote. Total fetal hemoglobin has increased and remained stable at between 23% and 43% for the five patients, who are producing “stably high” average amounts of fetal hemoglobin per F cell, at 10 to 16 picograms of fetal hemoglobin per cell, while 37% to 62% of the F cells’ total hemoglobin is fetal hemoglobin, she added. “The number of HbF-containing cells (F cells) was assessed by flow cytometry and the kinetics of F cell production was remarkably similar in all subjects.”.
The patients, who range in age from 12 to 26 years, are producing and maintaining very high numbers of F cells, or erythrocytes with measurable fetal hemoglobin, she said. Cell products were manufactured for 6 patients, Dr. Esrick reported, with cell doses of 3.3 to 8.3 million CD34-positive cells/kg and high vector copy numbers, “indicating successful manufacturing and a highly efficient vector.”. MDedge: Keeping You Informed. The information provided is for educational purposes only. This issue dives into the FDA's expedited review pathways, the progress and setbacks in gene therapy for hemophilia, and more. After testing and releasing the gene modified cells, the investigators infused them into the patients, who had received busulfan conditioning. The results of the pilot study of the shmiR vector approach, although preliminary and in need of longer follow-up, contribute to a larger body of research showing that multiple gene therapy approaches hold promise in this disease, said Robert Brodsky, MD, professor of medicine and director of the division of hematology at Johns Hopkins School of Medicine, Baltimore.
See our Other Publications. In a 3-patient study presented at ASH, investigators believe they have found a therapeutic target to cure sickle cell disease. Abstract #LBA-5. The only grade ≥3 AEs observed were related to central venous line (including thrombosis, pneumothorax, and infection). BCL11A regulates the fetal-adult hemoglobin switch by repressing expression at gamma (y)-globin locus, representing a potential therapeutic target for sickle cell.
There may soon be a cure for sickle cell disease. The patients are currently 7, 9, and 17 months post infusion. BCH-BB694 is an investigational gene therapy product manufactured by embedding a BCL11A-targeting structure in patients’ autologous CD34-positive cells using a shmiR lentiviral vector. “The transcription factor BCL11A is a strong repressor of gamma-globin, making it an appealing target for fetal hemoglobin induction.”. Gene therapy treatment also appeared to be associated with “consistent and substantial induction” of HbF. After gene therapy, the treated patients have not experienced any instances of vaso-occlusive crises, respiratory events, or neurologic events.
© 2020 MJH Life Sciences and HCPLive. “For all subjects, we estimated the fraction of RBCs containing significant Hb sickle polymers and the amount of polymer in each sickled RBC at physiologic oxygen tension (where 50% of monomeric hemoglobin was oxygen saturated, or the P50).”. Her coauthors reported disclosures related to Alerion Biosciences, Novartis, Orchard Therapeutics, Roche, AstraZeneca, and bluebird bio, among others. Abstract LBA-5.
BCL11A regulates the fetal-adult hemoglobin switch by repressing expression at the gamma (γ)-globin locus (Sankaran et al., Science, 2008), and thus it represents an appealing therapeutic target for sickle cell disease (SCD).BCH-BB694 is a lentiviral vector (LVV) encoding a shRNA targeting BCL11A embedded in a microRNA scaffold (shmiR) allowing erythroid-specific knockdown to induce γ …
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