Category REGENERATIVE MEDICINE

‘Organs in a dish’ pave the way for personalized medicine in gut and liver disease

Source Elsevier One of the most exciting advancements in stem cell research has been the development of organoid systems, which are organ-like three-dimensional structures that mimic their corresponding organ in vivo. In this important review in Digestive and Liver Disease, published by Elsevier, scientists highlight some of the established and exciting novel uses for organoids or

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Cell Therapy : Reviews and Industry News at June the 4th 2019

Reviews Gene Editing for Immune Cell Therapies Autologous T cells that have been genetically modified to express a chimeric antigen receptor targeting the B cell antigen CD19 have yielded remarkable clinical responses in patients with B cell malignancies, and are now on the market as anticancer ‘drugs’. [Nat Biotechnol] Abstract The Next Generation of CRISPR-Cas Technologies

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bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

ZYNTEGLO is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT) European marketing authorization for ZYNTEGLO follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval bluebird bio, Inc. announced today that the European Commission (EC)

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Research sheds light on new ways to target autoimmune disorders

Novel molecules developed by researchers at the Rockefeller University (NY, USA) have the potential to provide treatments for the millions of people who suffer from an autoimmune disorder. Reported recently in Nature Communications, Thomas Tuschl (Professor at Rockefeller University; NY, USA) and his fellow researchers from have developed small molecules that can bind to and inhibit one

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Regulatory intelligence: Update on regenerative medicine advanced therapies designations

This article discusses the scope and purpose of the special designation for Regenerative Medicine Advanced Therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT, such as keeping the US globally competitive in the field. They provide a tally of products receiving the special

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Using CAR T-cell Therapy in Older Patients With Aggressive Lymphoma

Article Highlights CAR T-cell therapy is gaining traction in relapsed/refractory lymphoma in older patients with aggressive disease. As a result of prospective trials investigating CAR T-cell therapy, there is potential that older patients with lymphoma can be spared multiple lines of chemotherapy and its resultant toxicities while still having long-term clinical benefit. Toxicities are still

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