Applications and the limitations of real-world data. Source Contract Pharma There are approximately 7,000 distinct rare diseases that exist affecting 350 million people worldwide, and approximately 80% of those rare diseases are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system1 have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies,
Penn to Broaden Pathways for Development of New CAR T Cell Therapies Following a landmark, first-of-its-kind collaboration that spawned the global CAR T cell therapy industry, the University of Pennsylvania and Novartis have concluded their seven-year research and development alliance, which resulted in the world’s first approved gene therapy for cancer. They will continue to
A new gene therapy turns glial cells — abundant support cells in the brain — into neurons, repairing damage that results from stroke and significantly improving motor function in mice. A paper describing the new therapy, which uses the NeuroD1 gene, appears online in the journal Molecular Therapy. Once further developed, this NeuroD1-based gene therapy potentially
Stem cell therapy, or stem cell transplant, is an emerging yet controversial treatment approach for multiple sclerosis (MS). While some data uphold it as one of the most efficacious MS treatments, to date there have been no controlled studies comparing it to conventional medicines and providing more robust evidence regarding its safety and clinical benefit. Under the topic
Penn study finds immunotherapy reduces cardiac fibrosis and restores heart function in mice after cardiac injury CAR T-cell therapy, a rapidly emerging form of immunotherapy using patients’ own cells to treat certain types of cancers, may be a viable treatment option for another life-threatening condition: heart disease. In a first-of-its-kind study, published today in Nature, researchers
The inhibitory receptors PD-1, Tim-3, and Lag-3 are highly expressed on tumor-infiltrating lymphocytes and compromise their antitumor activity. For efficient cancer immunotherapy, it is important to prevent chimeric antigen receptor T (CAR-T)-cell exhaustion. Here we downregulate these three checkpoint receptors simultaneously on CAR-T cells and that show the resulting PTL-CAR-T cells undergo epigenetic modifications and better control
Source Il Messaggero Matilde Leonardi, director of the Coma Research Center of the Besta Neurological Institute in Milan and a member of the Italian Society of Neurology, wants to be immediately clear. “The news about the former pilot is only fueling false hopes and deluding patients’ families.” Why is she so convinced that this therapy
The Italian biotech Genenta Science, a spin-out from San Raffaele Hospital has raised €13.2M in a private round to fund two ongoing phase I/II trials of its stem cell therapy Temferon for blood and brain cancer. Genenta Science will use the money to fund two European phase I/II trials, which the company launched in March. One
Tokyo Medical and Dental University(TMDU)-led study published in Nature shows that epidermal stem cells in the skin compete with each other to eliminate stressed, unfit cells; this maintains skin youthfulness, but with time also causes the skin to age. Aging is a process that results from the accumulation of damage due to failed repair and
Source https://www.sciencedirect.com/science/article/abs/pii/S002432051930757X?via%3Dihub&elqTrackId=b210a4797cc6484b94cf773fe330c747&elq=012dbc43f12c4386bdd3fa0ea93bb255&elqaid=26027&elqat=1&elqCampaignId=10598 Multiple sclerosis (MS) is a chronic autoimmune inflammatory disease of the central nervous system (CNS). In attempt to identify an appropriate treatment for improving the neurological symptoms and remyelination process, autologous and allogenic transplantation of mesenchymal stem cells (MSCs) have been introduced as an effective therapeutic strategy in MS. MSCs are a heterogeneous
