Of an estimated 6,500 to 7,000 known rare diseases, only a fraction – maybe 5% – have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams – including five new groups — of scientists, clinicians,
Interview with Dr. John Kennedy chief of the division of foot and ankle surgery and professor in the department of orthopedic surgery at NYU Langone in New York City. Here, Dr. Kennedy shares his insight into the future of platelet-rich plasma and stem cell therapy in sports medicine. Question: How do you see platelet-rich plasma
The number of cancer cell therapies has increased considerably over the last few years.1 There are several different types of cellular therapies, including chimeric antigen receptor (CAR) T cells, multiple tumor-associated antigen-specific T cells (TAA-T), natural killer (NK)-based therapies and T-cell therapies based on novel technologies like CRISPR.1 Amongst the different types of cell therapies in development,
Source Technologynetworks Since the gene-editing potential of CRISPR systems was realized in 2013, they have been utilized in laboratories across the world for a wide variety of applications. When this gene-editing power is harnessed with the proliferative potential of stem cells, scientists level up their understanding of cell biology, human genetics and the future potential
After receiving an adenoviral vector treatment of human ASM enzyme, transgenic mice with Niemann-Pick disease showed less accumulation of sphingomyelin, less damage to the brain and liver, and performed better in tests of motor function and memory. A new route for delivering gene therapy into the brain so that it is widely taken up without
The Alliance for Regenerative Medicine (ARM) and the National Association of Managed Care Physicians (NAMCP) Medical Directors Institute announced today the joint release of their recent study of medical director and manufacturer perspectives on value demonstration and reimbursement for cell- and gene-based regenerative and advanced therapies. The study, entitled “Roadmap for Navigating Cell and Gene
A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research. The study, “Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice,” appeared
Source NIH ‘Forward-oriented’ design might boost treatment effectiveness and broaden use. Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that delivers therapeutic genes—for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times
Gene therapies are getting approved. But there are major challenges ahead. A new report has been published by PwC Health Research Institute. After the thrill of approval has worn off and press coverage has faded, producers of gene therapies will be left with the challenge of getting their products to patients and getting paid for
Source ALS News Today Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease and Lou Gehrig’s disease, is a rare condition characterized by the degeneration of nerve cells that control voluntary muscles. The disease leads to gradually worsening symptoms that include muscle weakness, twitching, and stiffness. As more of these nerve cells, or motor neurons, are lost,
