BioCentury | Apr 1, 2020
Distillery Therapeutics

Gene editing to raise fetal hemoglobin levels for sickle cell disease

...DISEASE CATEGORY: Hematology INDICATION: Sickle cell disease Gene editing a promoter site controlling HBG1 and HBG2...
...disruption of the ZBTB7A binding site in the HBG1 and HBG2 promoter increased levels of HBG1...
...sickling in 69-79% of cells vs. 20% in unedited cells. TARGET/MARKER/PATHWAY: Hemoglobin subunit γ 1 (HBG1...
BioCentury | Dec 11, 2019
Tools & Techniques

Editas shows better gene editing using Cas9 alternative for sickle cell, thalassemia

...using an optimized Cas12a to disrupt the binding site for BCL11A within the HBG1 and HBG1...
...11A Cas9 - CRISPR-associated protein 9 Cas12a (Cpf1) - CRISPR from Prevotella and Francisella 1 HBG1...
BioCentury | Mar 20, 2019
Translation in Brief

Sangamo’s ZFN linkers finger new options

...some of which incorporated the new linkers, that targeted a 28-base pair sequence in the HBG1...
...of its products. Targets: Cas9 - CRISPR-associated protein 9 (Cas9); CEP290 - Centrosomal protein 290kDa; HBG1...
BioCentury | Dec 8, 2017
Targets & Mechanisms

Epiphenomena at ASH

...other four use it to induce therapeutic expression of fetal hemoglobin, which is encoded by HBG1...
...CRISPR Therapeutics AG, and another associated with CRISPR’s JV Casebia Therapeutics, targeted β-globin and the HBG1/HBG2...
...2 EZH1 - Enhancer of zeste homolog 1 EZH2 - Enhancer of zeste homolog 2 HBG1...
BioCentury | Jan 11, 2017
Clinical News

bluebird slips after latest LentiGlobin update

bluebird bio Inc. (NASDAQ:BLUE) lost $5.10 (11%) to $41.30 after reporting new results from three Phase I/II studies of LentiGlobin BB305 gene therapy , which the company is developing to treat sickle cell disease (SCD) and...
BioCentury | Dec 9, 2016
Company News

Cincinnati Children's Hospital, Calimmune deal

Calimmune gained rights to develop and commercialize ex vivo autologous gene therapies using the hospital's lentiviral hemoglobin subunit gamma (HBG) globin gene construct to treat sickle cell disease and beta thalassemia. Calimmune will combine the...
BioCentury | Nov 17, 2016
Translation in Brief

Editing hemoglobin

...team recapitulated the condition by editing two paralogous genes, hemoglobin subunits γ1 and 2 ( HBG1...
...disease, then used a lentiviral vector encoding Cas9 and a guide RNA to edit the HBG1...
BioCentury | Nov 10, 2016
Clinical News

LentiGlobin BB305: Additional Ph I/II HGB-206 data

Data from 7 evaluable patients with severe sickle cell disease in the open-label, U.S. Phase I/II HGB-206 trial showed that LentiGlobin BB305 led to a median vector copy number (VCN) of 0.08 vector copies/diploid genome...
BioCentury | Sep 22, 2016
Distillery Therapeutics

Therapeutics: Hemoglobin subunit γ 1 (HBG1); HBG2; CRISPR-associated protein 9 (Cas9)

...could help treat SCD. In HSPCs isolated from patients, a lentiviral vector encoding a CRISPR-Cas9-generated HBG1...
...mutant HBG1- or HBG2-expressing HSPCs in animal models of SCD. TARGET/MARKER/PATHWAY: Hemoglobin subunit γ 1 (HBG1...
BioCentury | Dec 21, 2015
Clinical News

Acetylon preclinical data

In rats and monkeys, once-daily oral ACY-957 increased hemoglobin subunit epsilon 1 ( HBE1 ; HBE ) and hemoglobin subunit gamma (HBG) globin mRNA, respectively, in peripheral blood. ACY-957 is a selective histone deacetylase 1 (HDAC1) and...
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