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
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)
Source: Nature Study of almost half a million people links mutation that protects against HIV infection to an earlier death. The scientist who edited the genomes of twin girls in an attempt to make them resistant to HIV might have inadvertently shortened their life expectancy. People with two disabled copies of the CCR5 gene — the version
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
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
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
Source Nature A glue ingredient was the secret to getting the mouse cells to multiply outside the body. Researchers have managed to grow large numbers of blood-forming stem cells in the lab using a surprisingly simple ingredient found in glue. And when injected into mice, the cells started producing key components of blood. “The finding
Source ARM Organovo Collaborates With MCRI and Leiden University Medical Center to Develop Stem Cell-Based Bioprinted Tissue Treatments for Kidney DiseaseMay 30, 2019 – (Organovo) – This multi-organizational effort integrates Organovo’s leading bioprinting platform with MCRI’s advanced stem cell differentiation technology and LUMC’s cell lines and clinical expertise. The collaboration has been made possible through generous
Encouraging Clinical Data Generated from Ongoing Proof-of-Concept Study Conducted by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget); Enrollment Expected to Complete in the First Half of 2020 Leverages Orchard’s Expertise in Treating Neurometabolic Diseases Using its Investigational Autologous Hematopoietic Stem Cell Gene Therapy Builds Upon Orchard / SR-Tiget Partnership to Develop Novel Gene Therapy Candidates Orchard Therapeutics, Fondazione
Universities of Birmingham and Oxford brings us a step closer to developing targeted therapies for inflammatory diseases. The research team shows, for the first time, that different types of fibroblasts – the most common cells of connective tissue in animals – are organised in different layers in the joint and are responsible for two very
