Penn Medicine researchers laud early results for CAR T therapy in lupus patients, point to broader horizon for the use of personalized cellular therapies Engineered immune cells, known as CAR T cells, have shown the world what personalized immunotherapies can do to fight blood cancers. Now, investigators have reported highly promising early results for CAR
Researchers find that combining novel gene-editing CRISPR technology with CAR T therapy could simplify and improve CAR T therapy in one fell swoop. Traditional CAR T Therapy A remarkable feat in cancer care, today people with difficult-to-treat blood cancers can receive CAR T therapy, a personalized “drug” made from their own immune cells. Chimeric Antigen Receptor T cell (CAR T)
Today, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. “Gene therapy for hemophilia has
The increasing integration of CAR T-cell therapies into the treatment paradigm for adult patients with relapsed or refractory multiple myeloma addresses several unmet needs and improves outcomes for this historically limited patient population. The increasing integration of CAR T-cell therapies into the treatment paradigm for adult patients with relapsed or refractory multiple myeloma addresses several
Applying regenerative medicine to a common shoulder surgery could have an impact on the need for follow-up revision surgery in some patients, according to a Mayo Clinic study of real-world evidence. Mayo Clinic researchers analyzed the largest set of data available to determine if adding bone marrow aspirate concentrate to repaired tissue after standard rotator cuff surgery would improve
A potential 1-time gene editing treatment for severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) is entering approval review by the US Food and Drug Administration (FDA), the European Medicines Agency, and the UK Medicines and Healthcare products Regulatory Agency, according to a company statement. Both diseases involve variants in the gene encoding β globin. The variants
OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency Sensorion, a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, announces that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to the Company’s lead therapy gene
A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and
Cambridge researchers are taking part in the world’s first clinical trial of red blood cells that have been grown in a laboratory for transfusion into another person. The manufactured blood cells were grown from stem cells from donors. The red cells were then transfused into volunteers in the RESTORE randomised controlled clinical trial. This is
In recent years, researchers have made measurable progress, using animal models, to promote tissue regeneration in spinal cord injuries (SCI) through implanted neural stem cells or grafts. Other efforts have shown that intensive physical rehabilitation can improve function after SCI by promoting greater or new roles for undamaged or spared cells and neural circuits. In
