Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol, new research shows. That’s because the shape and structure of the phytosterols help the genes get where they need to be inside cells. The findings by Oregon State University researchers, published today in Nature Communications, are important because many illnesses that
If approved, 1st Gene Therapy in U.S. for the Treatment of Any Type of Hemophilia BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia
Industry leaders, patient advocates, researchers unite to maximize the incredible potential of transformative gene therapies The Institute for Gene Therapies (IGT) launched today with a focus on advocating for a modernize the U.S. regulatory and reimbursement framework so that gene therapies can deliver their significant potential to patients. IGT will educate stakeholders across the healthcare
A team of scientists from Purdue University and institutions around the world have come together to better understand the growing number of worldwide patented innovations available for gene therapy treatment. The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases. Now, a team of scientists
In the first clinical study of its kind in the US, researchers used CRISPR to modify CAR T cells to make them more potent against cancer, but the clinical benefits are unknown. mmune cells altered by CRISPR are safe to use in cancer patients, according to results of a clinical trial reported February 6 in Science.
The University of Hertfordshire and the Cell and Gene Therapy Catapult (CGT Catapult) have announced the launch of a new course specifically addressing the foreseeable skills gap in the manufacture of cell and gene therapies as they progress towards manufacturing at scale. Developed in a collaboration between the two organisations, this three-day course will provide
Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum München) have developed a gene therapy
Within the framework of an international study, Lund University and Skåne University Hospital have started treating patients with hemophilia with gene therapy, something that began in January this year. The hope is that the new treatment will significantly simplify everyday life for those with severe hemophilia. Hemophilia is a genetic disease where the body does
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
