The interim Phase I trial data was only from 35 people, but results look good so far. An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial. Researchers running a Phase I safety trial for the drug,
Researchers are testing CAR T cell therapy as a way to reset the immune system in lupus, Graves’ disease and other conditions where the body’s defenses go rogue At age 49, Jan Janisch-Hanzlik’s multiple sclerosis was destroying her freedom to live the life she wanted. She gave up her active nursing job for a desk
Stem-cell memory T (TSCM) cells are a rare subset of immune cells with the ability to self-renew, persist long term, and mount potent anti-tumor responses. These properties make them an attractive candidate for next-generation CAR T-cell therapies. However, their clinical potential has not previously been demonstrated in humans. An international team of researchers co-led by
A phase 2 study published in the Journal of Clinical Oncology shows that a stem cell product called dilanubicel safely enabled umbilical cord blood transplants in 28 patients A new way of using umbilical cord blood for treating blood diseases could make the treatment more accessible to patients who need a stem cell transplant. A
Groundbreaking AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound and profound hearing loss The U.S. Food and Drug Administration approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. Otarmeni is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any
The third Clinical Trials Plenary Session at the AACR Annual Meeting 2026 was dedicated to “Cellular Therapies and Complex Immunotherapies” and provided updates on sophisticated immunotherapeutic CAR-T cells strategies, with special emphasis on their application for solid tumors. Can CAR T-cell therapy intercept disease in patients with smoldering myeloma? The first presenter Omar Nadeem, MD, of
In a breakthrough for gene therapy, the international academic journal Nature has published a landmark clinical study detailing the successful use of a novel base-editing drug to treat β-thalassemia. Jointly conducted by ShanghaiTech University, the First Affiliated Hospital of Guangxi Medical University, Fudan University, and CorrectSequence Therapeutics, the early-stage clinical trial of the base-editing drug CS-101 injection reports that
For the first time, scientists have used a modern cell therapy called CAR-T to treat a patient with three different life-threatening autoimmune diseases that had resisted years of treatment. The patient, who once required daily blood infusions, has been in remission without needing additional treatment for a year since the CAR-T therapy. The case report,
Gene edits could solve obstacles to treatment for the most common types of cancer Since 2017, a personalized immunotherapy called Chimeric Antigen Receptor, or CAR-T cell treatment, has worked wonders to treat patients with blood cancers such as leukemia. But when it comes to treating solid tumor cancers, such as lung, breast and kidney cancers
New findings suggest blocking a key sensor protein could help neurons repair damaged connections Researchers from the Icahn School of Medicine at Mount Sinai have discovered a molecular switch in neurons that limits the regrowth of damaged axonal fibers. The findings, published in the journal Nature, show that blocking a protein called the aryl hydrocarbon receptor (AHR) may
