Los Angeles, CA – The gene SYNGAP1, the variants of which are top risk factors for Autism Apectrum Disorder (ASD), has previously unappreciated effects on the developing brain, according to a new study published in Nature Neuroscience. The study shows how disease-causing variants of SYNGAP1, thought primarily to affect synapses between mature neurons, could disrupt...
research News
SAN FRANCISCO and SUZHOU – Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, and IASO Biotherapeutics (IASO Bio), today jointly announce that the Center for Drug Evaluation (CDE) of China’s National Medical Products...
Baltimore, Maryland – The Bespoke Gene Therapy Consortium, a public-private collaboration involving the National Institutes of Health, FDA, industry and patient groups intended to help accelerate the delivery of AAV-based gene therapies for rare diseases, is making slow but steady progress, a representative said at the 2024 American Society of...
Cambridge, UK – A combination of two drugs could improve outcomes and reduce the need for toxic chemotherapy for B-cell acute lymphoblastic leukemia (B-ALL), the commonest cancer in childhood and one that can be particularly difficult to treat in older patients, according to Cambridge scientists. Although the research has so...
Madrid, Spain – A few years ago, a patient was cured of multiple myeloma after being treated for hepatitis C, astounding researchers from the group led by Joaquín Martínez, from the H12O-CNIO Haematological Tumours Clinical Research Unit, a collaboration between the Hospital 12 de Octubre (H12O) and the National Cancer...
Washington, DC – Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center for Biologics...
Basel, Switzerland – Some pathogens hide inside human cells to enhance their survival. Researchers at the University of Basel, have uncovered a unique tactic certain bacteria use to spread in the body without being detected by the immune system. In their study, they reveal the crucial role of a bacterial...
TOKYO, Japan — Astellas Pharma Inc. President and CEO Naoki Okamura contributed to the development of a pediatric formulation to treat schistosomiasis as a member of the Pediatric Praziquantel Consortium. The Consortium announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted...
NORTH BETHESDA, Md. — The Foundation for the National Institutes of Health (FNIH) announces the launch of a public-private partnership to build the largest data source for amyotrophic lateral sclerosis (ALS) and expedite the identification of biomarkers and clinical outcome assessments that would allow earlier diagnosis and accelerated drug development...
EAST LANSING, Michigan – Barth syndrome (BTHS) is an ultra-rare but debilitating genetic disease affecting cell mitochondria. High infant mortality due to cardiac complications and a weakened immune system significantly decrease life expectancy. Current medications treat symptoms but not the cause, leaving an unmet clinical need to develop effective treatments...