NEW YORK – Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced results of a well-controlled non-human primate study, which demonstrate that route of adeno-associated virus (AAV) administration plays a critical role in optimizing gene therapy delivery to key...
research News
Indianapolis, Ind. – Alois Alzheimer recognized more than a century ago the role of neuroinflammation in the development of the disease that would bear his name. “If you actually go back to his original drawings, he was drawing the cells, which today we know are microglia,” immune cells of the...
WUHAN, China and SAN DIEGO, Calif. — Neurophth Therapeutics, Inc. announced today that the last patient has been enrolled in Phase I/II clinical trial of Opvika® (Esonadogene Imvoparvovec) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON). Neurophth is conducting a Phase I/II, single-arm, multi-center study aimed...
Jerusalem, Israel – In a compelling Genomic Press Interview published in Brain Medicine on September 25, 2024, Professor Hermona Soreq of the Hebrew University of Jerusalem in Israel unveils the profound implications of her groundbreaking research on the cholinergic system and small RNA regulators in brain-body communication. Prof. Soreq, holder...
Toronto, Canada – Researchers at The Hospital for Sick Children (SickKids) have found that inflammation in an immune cell may be responsible in part for some severe symptoms in a group of rare genetic conditions called lysosomal storage diseases (LSDs). LSDs affect about one in 7,700 live births worldwide. Children...
TORONTO, Canada – A new round of clinical trials supported by the Ontario Institute for Cancer Research (OICR) will harness a unique opportunity to test some of the newest treatment strategies for a range of different cancers. OICR has announced two new clinical trials as part of its Window-of-Opportunity (WOO)...
New Rochelle, NY – Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy. They also showed that after single dose inhaled delivery of SP-101, SP-101 vector genomes were detected throughout the respiratory tract...
Barcelona, Spain – Researchers at the Centre for Genomic Regulation (CRG), the University of the Basque Country (UPV/EHU), Donostia International Physics Center (DIPC) and the Fundación Biofisica Bizkaia (FBB, located in Biofisika Institute) have developed an artificial intelligence which can differentiate cancer cells from normal cells, as well as detect...
Liverpool, UK – In people with a rare condition called light chain amyloidosis, light chain proteins—which are a component of antibodies—mutate and build up in different organs. In new research published in The FEBS Journal, investigators have identified and characterized an antibody fragment that can bind to abnormal light chains...
London, UK – Antibody treatment which activates the patient’s own immune system against cancer, known as immunotherapy, is increasingly being investigated as an alternative for chemotherapy and radiotherapy. This is because it specifically targets the cancer cells, which reduces the side effects seen with more conventional therapies. Tumours, such as...