The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials.
First agreements with statutory health insurances utilize bluebird’s innovative value-based payment model and provide coverage for ZYNTEGLO for up to 50% of patients in Germany First qualified treatment center established at University Hospital of Heidelberg to provide ZYNTEGLO to patients bluebird bio, Inc. announced the launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a
ARM today announced the release of a report, “A Transformative Therapy Value Model for Rare Blood Diseases.” The report uses a refined value model to assess the impact of durable and potentially curative cell and gene therapies on health system costs. Produced in collaboration with the Marwood Institute, this report calculates the potential cost savings that
Johns Hopkins researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a “suicide gene” to pediatric brain tumor cells implanted in the brains of mice. The poly(beta-amino ester) nanoparticles, known as PBAEs, were part of a treatment that also used a drug to kill the cells and prolong the test
Scientists have discovered how to switch off a key ‘pain gene’, dramatically raising hopes of a long-term treatment to relieve the agony of serious illness for millions. The revolutionary technique alters a patient’s DNA, silencing a gene that transmits pain signals up the spine. Preliminary studies on mice have already proven successful and US researchers
Researchers are moving closer to a new approach for improving spinal fusion procedures and repairing broken or defective bones that avoids an over-production of bone that commonly occurs in current treatments. In a preclinical study, researchers significantly reduced undesired bone growth outside of targeted repair areas in rat femurs by delivering a potent bone-forming protein
The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many
Mayo Clinic offers a unique regenerative medicine approach for repairing knee cartilage, which can be completed in a single surgery. The Food and Drug Administration approved the use of this technique, known as recycled cartilage auto/allo implantation (RECLAIM), in a trial utilizing the stem cell bank in the Mayo Clinic Center for Regenerative Medicine. “Mayo
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A published Gene therapy with valoctocogene roxaparvovec reduced annualized rates of bleeding events and led to discontinuation of prophylactic factor VIII among patients with hemophilia A, according to results of a prospective study with multiyear follow-up published in The New England Journal of Medicine. “The treatment burden is
Novel spinal therapy/delivery approach prevented disease onset in neurodegenerative ALS disease model in adult mice and blocked progression in animals already showing disease symptoms Writing in Nature Medicine, an international team headed by researchers at University of California San Diego School of Medicine describe a new way to effectively deliver a gene-silencing vector to adult amyotrophic
