bluebird bio presents positive results for inherited blood disorder gene therapies

06 Nov 2023
www.pmlive.com
Gene TherapyPriority ReviewASH
bluebird bio has announced updated results from its gene therapy programmes in sickle cell disease (SCD) and beta-thalassaemia (beta-thal).
The results, which will be presented at the American Society of Hematology (ASHsickle cell disease (SCD)sitiobeta-thalassaemia (beta-thal)p data from the company's lentiviral vector (LVV) gene therapies in SCD patients who have a history of vaso-occlusive events (VOEs) and those with beta-thalassemia who require regular red blood cell transfusions.
Affecting approximately 100,000 people in the US, SCD is a life-long, incurable genetic disease that causes red blood cells to take a distinct crescent shape, which can block blood vessels and affect the waSCDxygen is carried around the body.beta-thalassemia
Bluebird’s lovotibeglogene autotemcel (lovo-cel) iSCDn investigational one-time treatment designed to add functional copies of a modified form of the beta-globin gene into a patient’s own haematopoietic stem cells so their red blood cells can produce anti-sickling haemoglobin.
Updated reslovotibeglogenevo-cel-treated patients followed for a median of 35.5 months show that the therapy, which was accepted by the US Food and Drbeta-globinration for priority review earlier this year, was associated with sustained adult haemoglobin production and near-complete resolution of VOEs and severe VOEs, as well as sustained improvements in health-related quality of life.
Beta-thal, another inherlovo-celod disorder, is caused by a gene defect that impairs the ability of red blood cells to produce haemoglobin.Food and Drug Administrationre form of beta-thal develop life-threatening anaemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks.
Beta-thalgene autotinherited blood disorderis already approved in the US under the brand name Zynteglo for adult and paediatric patients with beta-thal who require regular red blood cell transfusions, works in a sanaemiaway to lovo-cel to allow patients to make normal-to-near-normal levels of total haemoglobin without regular red blood cell transfusions.
Betibeglogene autotemcel (beti-cel) to be presented at ASH demonstrate a sustained transfusionZynteglodence after up to nine years of follow-up and improvements in quality of life reported at month 36.lovo-cel
Richard Colvin, bluebird’s chief medical officer, said: “The robustness of this dataset is unparalleled, giving us continued confidence that the transformational impact these therapies may have for patients is sustained over time.
“We especially look forward to the first ever presentation of data from our long-term follow-up study of lovo-cel, the most deeply studied gene therapy in development for SCD.”
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Targets
Drugs
AI Agents Built for Biopharma Breakthroughs
Accelerate discovery. Empower decisions. Transform outcomes.
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.