A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body. The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that cause a rare condition called Leber’s congenital amaurosis 10 (LCA10).
ARM today released its 2019 Annual Report and Sector Year in Review, highlighting the organization’s key priorities and initiatives, as well as offering an in-depth look at trends and metrics for the cell therapy, gene therapy, and tissue engineering sector. ARM, which celebrated its 10th anniversary in 2019, is the premier international advocacy organization representing the cell
The Canadian government is investing almost $7 million in stem cell research and regenerative medicine. William Amos, Parliamentary Secretary to the Minister of innovation, Science and Industry, was at Maisonneuve-Rosemont Hospital Monday to announce that $6.9 million would go to the Stem Cell Network’s competitive research funding program. “Stem cell and regenerative medicine are a
Researchers use lipids to safely deliver gene therapy to the eye, successfully holding off advance of rare, inherited eye disorder Researchers at Case Western Reserve University have used a unique method to safely deliver gene therapy to fight a rare, but irreversible, genetic eye disorder known as Stargardt disease. By using chemically modified lipids—instead of the
Gene therapy to treat Huntington’s disease Huntington’s disease (HD) is a rare disease characterized by abnormal chorea movement and caused by Huntingtin (Htt) gene mutation and neurodegeneration in a brain area called striatum. A research group led by Dr. Gong Chen, a former professor at Penn State University and now leading a brain repair center
The excitement about cell and gene therapies is almost tangible within the biotech and pharma industry. Over 950 companies are actively developing advanced therapies, which are expected to make exceptional improvements to peoples’ lives in the next decade. Although hopes are high, the industry still faces a number of challenges in cell and gene therapy manufacturing, mainly
Chimeric antigen receptor (CAR) T-cell therapy may keep advanced blood cancers at bay, but it can’t keep the wolf from the door, warn experts on healthcare policy and financing. For all the talk about the steep costs of CAR T-cell therapy and how to pay for it, comparatively little attention has been paid to the
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
Neel Anand, MD, serves as professor of orthopedic surgery and director of spine trauma at the Cedars-Sinai Spine Center in Los Angeles. Dr. Anand has dedicated his practice to the advancement of minimally invasive spine surgery and served as principal investigator in several FDA clinical trials. Here, Dr. Anand discusses the hype around stem cells and
