The new therapy was generally well-tolerated and prevented key signs of the condition in animal models. Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities. Angelman
This review aims to outline the particular approaches in the different published clinical trials for cell-based therapies for retinal diseases. The retinal pigment epithelium (RPE) is implicated in the pathophysiology of many retinal degenerative diseases. This cell layer is also an ideal target for cell-based therapies. Several early phase clinical trials evaluatingcell therapy approaches for
Is there enough evidence to show Biogen’s new Alzheimer’s drug Aduhelm provides a benefit to patients, physicians and society beyond the current standard care? The answer from a group of ICER experts, perhaps unsurprisingly, was “no.” During a daylong meeting Thursday that featured debate over the drug’s cost and more, experts voted 15 to 0 that
EMA’s safety committee, PRAC, has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known as acute myeloid leukemia (AML). The European Medicines Agency’s (EMA’s) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known
Sickle cell disease is the most common deadly genetic disorder. It results from a mutation that causes normally pliable red blood cells to become brittle and sickle shaped. This leads to pain, organ damage and early death. A team of scientists led by Mitchell J. Weiss, MD, PhD, and Jonathan Yen, PhD, at St. Jude, and David Liu,
St. Jude Children’s Research Hospital provides the first gene therapy manufacturing and distribution cost report for hemophilia B. A St. Jude Children’s Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene therapy now in development. The study appeared
Peter MacCallum researchers are developing a potential new way to make CAR T-cell therapy more effective against breast cancer and other solid cancers. CAR T-cell therapy is a type of immunotherapy where a patient’s own immune cells are collected and reengineered, before being infused back into the patient to fight their cancer. But CAR T-cells
The approvals of the first gene and cell therapies in recent years have ushered in a new era of medicine. These cutting-edge approaches are poised to change the trajectory of how we treat cancer, fatal genetic conditions, and other devastating diseases. But these innovations also come with their own set of challenges, including issues of
Deep Pharma Intelligence released a special 115-page analytical report Cell Therapies In Healthcare Landscape Overview 2021 (Stem Cells and CAR-Ts), which is the first comprehensive and systematic overview of cell therapies landscape by the company. This report is specifically focused on Stem Cell therapies and Chimeric Antigen Receptor T-cell (CAR-T) therapies, and it is the first
Immunotherapies that fight cancer have been a life-saving advancement for many patients, but the approach only works on a few types of malignancies, leaving few treatment options for most cancer patients with solid tumors. Now, in two related papers published April 28, 2021, in Science Translational Medicine, researchers at UCSF have demonstrated how to engineer smart immune cells
