The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an
Researchers at IRB Barcelona reveal that vitamin B12 significantly boosts the efficiency of cellular reprogramming, thus holding promise for regenerative medicine. Vitamin B12 supplementation shows potential in speeding up tissue repair in a model of ulcerative colitis—an observation that points to potential new treatments for inflammatory diseases. The discovery has been published in the journal Nature
New research at ACR Convergence 2023, the American College of Rheumatology’s (ACR) annual meeting, shows that patients with refractory juvenile systemic sclerosis improved significantly on nearly all measures for two years following autologous stem cell transplant (Abstract #L06). Juvenile-onset systemic sclerosis (jSSc), also called scleroderma, is a disfiguring autoimmune disorder marked by hardening of the skin
A single infusion of a CRISPR-based gene editing therapy significantly reduced LDL-C and PCSK9 levels in patients with heterozygous familial hypercholesterolemia (HeFH), based on findings from the VERVE-101 trial presented Nov. 12 at AHA 2023. In presenting the findings, Andrew Bellinger, MD, PhD, said they provide the first proof-of-concept for in vivo DNA base editing in humans.
Scientists have long sought to understand how certain animals, such as zebrafish, are able to regrow nerve fibers and regain lost motor function after spinal cord injury. In humans, these injuries are irreparable and result in permanent loss of function, such as paralysis. An international team led by Daniel Wehner of the Max Planck Institute
Characterized by extensive damage to joints and debilitating pain, osteoarthritis (OA) impacts millions of people worldwide and has long posed a substantial clinical and economic burden. In spite of advances in diagnosis, medications, and short-term pain management solutions, the elusive goal of a disease-modifying OA drug has remained out of reach. In recent years though,
The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S. since late last year as the FDA sought more details from Verve. Verve Therapeutics plans to soon start testing a gene editing therapy for heart disease in the U.S. after federal regulators lifted an order that had
Researchers are testing an approach that involves replacing a mutated gene in the inner ears of children with severe hearing loss. Two companies have launched clinical trials to see if they can restore hearing to children with a rare type of genetic deafness. Akouos and Decibel Therapeutics, both based in Boston, are testing experimental therapies in
Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months. Immunotherapies, which mobilize the body’s immune defenses against a cancer, have not been effective for GBM, in part because the tumor’s surrounding environment is largely impenetrable to assaults from the body’s immune system.
Incredibly detailed cell maps help pave the way for new generation of treatments A group of international scientists have mapped the genetic, cellular, and structural makeup of the human brain and the nonhuman primate brain. This understanding of brain structure, achieved by funding through the National Institutes of Health’s Brain Research Through Advancing Innovative Neurotechnologies® Initiative, or
