BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1–2 dose-escalation study. METHODS Was conducted an open-label, single-group, multicenter, phase 3
Immunotherapy is increasingly becoming a successful way to treat cancer. Researchers at Uppsala University have now developed armed CAR-T cells that reinforce the immune defense against cancer and that could increase the possibilities to successfully treat solid tumors. The study has been published in the journal Nature Biomedical Engineering. The use of immunotherapy to treat cancer
Researchers at Weill Cornell Medicine have identified a key protein that induces the program to build specialized liver blood vessels. The discovery could lead to engineered replacement hepatic tissue to treat common liver diseases. There are many types of blood vessels in the human body that are functionally different from each other. In the liver,
Findings shine light on underpinnings of COPD, pave new direction for future research on treatments A new type of cell that resides deep within human lungs and may play a key role in human lung diseases has been discovered by researchers at the Perelman School of Medicine at the University of Pennsylvania. The researchers, who
MIT spinout Frequency Therapeutics’ drug candidate stimulates the growth of hair cells in the inner ear. Most of us know someone affected by hearing loss, but we may not fully appreciate the hardships that lack of hearing can bring. Hearing loss can lead to isolation, frustration, and a debilitating ringing in the ears known as
A new approach allowed scientists to alter immune cells within 24 hours versus the standard of up to two weeks A new approach from Penn Medicine researchers could cut the time it takes to alter patients’ immune cells for infusion back into the body to find and attack cancer. The cell manufacturing process for this type of
Researchers from North Carolina State University and the University of North Carolina at Chapel Hill have developed an implantable biotechnology that produces and releases CAR-T cells for attacking cancerous tumors. In a proof-of-concept study involving lymphoma in mice, the researchers found that treatment with the implants was faster and more effective than conventional CAR-T cell
Chimeric antigen receptor (CAR) T cells can be remarkably effective in treating leukemias and lymphomas, but there are no successful immunotherapies for neuroendocrine tumors (NETs) and gastrointestinal cancers (GICs) yet. Researchers at Penn Medicine have discovered that CAR-T cells directed to a tumor antigen, CDH17, a cell surface marker expressed on both NETs and GICs but also
A novel new therapy has been found to reduce harmful plaque in arteries and change its composition so it is less likely to rupture and cause a heart attack, following a clinical trial led by the Victorian Heart Institute (VHI) at Monash University. The HUYGENS study treated high-risk patients over a 12 month period and
New opportunities towards gene therapy and diagnosis for the blinding eye disease, retinal dystrophy, may now become available following work done by the Eye Genetics Research Unit at Children’s Medical Research Institute. This work was published in the Journal of Personalized Medicine today. The team looked at the RPGR gene which is involved in maintaining
