Category REGENERATIVE MEDICINE

American Academy Neurology issues Evidence in Focus article on Duchenne muscular dystrophy gene therapy

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

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World’s first patient treated with personalized CRISPR gene editing therapy through CHOP and Penn Med collaboration

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

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Enhanced CAR T cell therapy offers new strategy for lymphoma

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

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New CAR T cell therapy benefits patients with advanced thyroid cancers

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

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FDA clears IND for clinical trial testing switchable CAR-T therapy in patients with autoimmune diseases, without chemotherapy

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

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Innovative gene therapy developed to treat a rare and devastating genetic heart disease in young men

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),

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Major advances in the treatment of type 1 diabetes

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

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Cultured tissue from nasal cartilage cells helps with complicated knee injuries

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

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Gene therapy in children with AIPL1-associated severe retinal dystrophy can improve vision

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

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