Penn study shows delivering a “distant cousin” of a key protein prevented muscle damage without triggering an immune response in large animal models A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal
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,
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 BioProcess International Over 20 cell and gene-based therapies are expected to be filed or to receive approval decisions over the next 18 months, according to the Alliance for Regenerative Medicine (ARM). Earlier this year, the US Food and Drug Administration (FDA) laid out its action plan to deal with the large upswing in the
Source: Life Science Leader Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient. Exhibit A: Novartis’ Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment, priced at $2.125 million, is the world’s most expensive drug to date. But cell and gene therapies are their own animals; the differences between
