Results from the Phase 2 RedirecTT-1 study demonstrate deep responses with 78.9 percent overall response rate through dual targeting of GPRC5D and BCMA Data signal potential of novel, off-the-shelf approach in patients with extramedullary disease who face significant unmet needs MILAN, Italy — Johnson & Johnson (NYSE: JNJ) announced today new results from...
treatment News
JERSEY CITY, N.J. — Tanabe Pharma America, Inc. (TPA) today announced the presentation of exploratory preclinical findings into edaravone’s activity in an ALS-relevant cell model. The data will be shared during the Motor Neurone Disease Association (MNDA) 36th International Symposium on ALS/MND, taking place in San Diego, California, from December 5-7, 2025...
SAN DIEGO, CA ― The targeted therapy bezuclastinib was safe and rapidly reduced markers of disease burden while also improving symptoms for patients with a rare blood disorder called nonadvanced system mastocytosis, according to results of the Phase II SUMMIT trial reported by researchers at The University of Texas MD...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of a manuscript, “Gene Therapy in the Putamen for Curing AADC Deficiency and Parkinson’s Disease,” in the European Molecular Biology Organization Journal. The paper describes a pioneering approach that delivers gene therapy to a specific part of the brain...
OSLO, Norway – Targovax ASA (OSE: TRVX), a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors, today announce that the European Patent Office has granted EU Patent no EP3402889. The patent covers the use of ONCOS-102 in combination with chemotherapy in malignant pleural mesothelioma. Torbjørn Furuseth,...
Basel, June 5, 2009 – New data show that at 12 months newly diagnosed patients taking Tasigna® (nilotinib) to treat a life-threatening form of chronic myeloid leukemia (CML) had rapid responses and a deep reduction in the amount of the abnormal protein that causes this disease. “Tasigna now demonstrates potential...
DALLAS, Texas — Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to...
CTA approved by Health Canada in November 2021; IND approved and open in 2019 Queen’s University selected as initial clinical site under the direction of Dr. Jagdeep Wahlia, principal investigator PPT1 enzyme activity of 5% or greater normalizes survival and significantly improves clinical phenotype based on natural history data Preliminary...
DALLAS, TX — Taysha Gene Therapies, Inc., a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that it will present clinical data on its TSHA-102 program in evaluation for Rett syndrome during...
DALLAS – Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced publication of new preclinical data for TSHA-104 in...