This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more
Treatment uses person’s own stem cells instead of donor cells UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and
Identification of GPR108 as ‘lock’ for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapies Researchers led by a team at Massachusetts Eye and Ear have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types–the most commonly
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
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
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
