Category ADVANCED THERAPY MEDICINAL PRODUCT

First successful treatment of severe pulmonary hypertension using umbilical cord stem cells

Hannover Medical School doctors successfully treat three-year-old girl / Publication in “Nature Cardiovascular Research“ Clinical researchers at Hannover Medical School (MHH) have succeeded for the first time worldwide in stopping the usually fatal course of the disease in severe pulmonary hypertension thanks to a novel therapeutic approach. A three-year-old girl suffering from so-called pulmonary arterial

Read More


Recent progress in cell therapy research: a review in Nature outlines engineering disciplines, genome and epigenome editing, synthetic biology and biomaterial-mediated immune modulation.

Given recent progress in cell therapy research, it is clear that the engineering disciplines outlined in this Review will play an increasing role in the creation of new product pipelines with improved safety, efficacy and accessibility for patients. Recent scientific advances have not only demonstrated the potential impact of technologiesdeveloped by each of these fields,

Read More


Financial risks posed by unproven cell interventions in Japan

The research group led by Professor Misao Fujita conducted a study to estimate the total amount of refund the Japanese government would pay if a patient received unproven cell interventions and applied for a medical expense deduction. Some regenerative medicines whose safety and efficacy have not been fully confirmed in clinical trials and other studies are offered

Read More


Cell-Derived Exosome Therapy May Help Repair Abnormal Heart Rhythm

Cedars-Sinai Investigators Found It Also Reduced Scarred Heart Tissue in Animals Vesicles secreted from human heart cells may repair damaged tissue and prevent lethal heart rhythm disorders, according to a new study from investigators in the Smidt Heart Institute at Cedars-Sinai. The research, published in the European Heart Journal, could lead to a new way to

Read More


Cell Treatment Slows Disease in Duchenne Muscular Dystrophy Patients

Cell Therapy Developed by Smidt Heart Institute Leader Delays Disease Progression in Patients Who Have Few Treatment Options A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles–including the heart muscle–in Duchenne muscular dystrophy patients, a new study published in the international peer-reviewed journal The Lancet shows. If the HOPE-2 study’s

Read More


Natural Killer Cells Complexed With a Bispecific Antibody May Provide New Treatment Option for Patients With Advanced Lymphoma

Natural killer (NK) cells derived from cord blood that were activated and complexed with a CD30/CD16A bispecific antibody elicited an 89 percent overall response rate in patients with relapsed or refractory CD30+ lymphoma, according to results from a phase I/II clinical trial presented at the AACR Annual Meeting 2022, held April 8-13. “We were favorably surprised by the

Read More


Foreseeing the future of European medicine

Experts say careful application of advanced tech could usher in a golden age of healthcare New technologies could enable medicine to progress in leaps and bounds, but only with the right regulatory and ethical frameworks. That was one of the messages from panelists discussing the future of medicine at the Science|Business conference Horizon Europe: The first assessment as

Read More


Gene Therapies Developers slowly emerge from a pandemic: a new ebook published by BioProcess Insider

This eBook gauges shifting expectations for the gene therapy industry amid the COVID-related uncertainties and clinical setbacks of the past couple years. BioProcess Insider founding editor Dan Stanton reports on the January 2022 Phacilitate Advanced Therapies Week event, specifically a standing presentation on the 10 most important industry drivers from the past year. Since 2017, advancements in

Read More


Gene therapy in transfusion-dependent beta-thalassemia

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine. The therapy represents a potentially curative treatment option for patients who must otherwise rely on life-long red blood cell transfusions. This approach provides

Read More


Landing therapeutic genes safely in the human genome

Researchers at Harvard’s Wyss Institute, Harvard Medical School, and the ETH Zurich predict and validate genomic safe harbors for therapeutic genes, enabling safer, more efficient, and predictable gene and cell therapies. Many future gene and cell therapies to treat diseases like cancer, rare genetic and other conditions could be enhanced in their efficacy, persistence, and

Read More