To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology®. AAN Evidence in Focus articles highlight the strength of the current evidence for
A landmark study from CHOP and Penn Medicine showcases the power of customized gene editing therapy to treat a patient with a rare metabolic disease In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children’s Hospital of
Next-generation CAR T cell therapy works where other CAR T cell therapies have failed A next-generation “armored” CAR T cell therapy showed promising results in a small study of patients whose B-cell lymphomas continued to resist multiple rounds of other cancer treatments, including commercially available CAR T cell therapies. The new therapy diminished cancer in 81 percent of patients
A new chimeric antigen receptor (CAR) T cell therapy called AIC100, which targets the ICAM-1 protein, demonstrated encouraging responses and an acceptable safety profile in patients with two types of advanced thyroid cancer, according to researchers from The University of Texas MD Anderson Cancer Center. Results from the first-in-human Phase I trial were presented today at the American Association
Innovative cellular therapy has potential to treat patients with lupus, systemic sclerosis, myositis and RA without chemotherapy-induced immune suppression. Calibr-Skaggs Institute for Innovative Medicines, the drug discovery division of Scripps Research, announced today that the FDA has cleared their investigational new drug (IND) application to study switchable chimeric antigen receptor T cell (sCAR-T) therapy (CLBR001
A CNIC team has designed a gene therapy-based treatment that could transform the approach to arrhythmogenic cardiomyopathy type 5 (ARVC5), a rare and fatal genetic disease that particularly affects young men. A team from the National Center for Cardiovascular Research (CNIC) has developed an innovative gene therapy that could transform the treatment of arrhythmogenic cardiomyopathy type 5 (ARVC5),
Recent decades have seen a lot of progress in diabetes treatment. In a new study, Per-Ola Carlsson, Professor of Medical Cell Biology, has obtained promising results. “This is the first time anyone has succeeded in transplanting insulin-producing cells into another individual without immunosuppressive drugs,” he says. The treatment available for type 1 diabetes since the
Damage to joint cartilage is painful and limits mobility. Researchers at the University of Basel and the University Hospital of Basel are therefore developing cartilage implants from cells from the nasal septum. A recent study shows that a longer maturation time for the cultured cartilage brings a significant improvement even in cases of complicated cartilage
Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with AIPL1-associated severe retinal dystrophy. This is the first human trial of gene supplementation therapy targeting this condition. Retinal dystrophy caused by biallelic variants in the AIPL1 gene
An 18-year survivor of neuroblastoma, a solid childhood tumor that develops in immature nerve cells, is believed to represent the longest reported remission to date in a patient treated with chimeric antigen receptor (CAR) T cell therapy. The findings, published in Nature Medicine, describe several long-term survivors in a follow-up from a phase 1 clinical trial
