LARKSPUR, Calif. and STAMFORD, Conn. — The Global Coalition for Adaptive Research (GCAR) and Purdue Pharma L.P. (Purdue) today announced the activation of investigational tinostamustine in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), a pioneering, international adaptive platform trial designed to accelerate the identification of effective treatments for...
treatment News
Boston, Mass. – Positive high-level results from a global, randomised, double-blind, placebo-controlled Phase III trial in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalised myasthenia gravis (gMG) showed that gefurulimab met its primary and all secondary endpoints. Data demonstrated a statistically significant and clinically meaningful improvement from baseline in Myasthenia...
Gemcitabine plus cisplatin significantly improved overall survival (OS) when compared with fluorouracil plus cisplatin in treatment-naïve patients with advanced nasopharyngeal carcinoma, according to final results of a phase 3 study published in the Journal of Clinical Oncology.¹ The GEM20110714 trial (ClinicalTrials.gov Identifier: NCT01528618) enrolled 362 patients who were randomly assigned 1:1...
DRESDEN, Germany – GEMoaB, a biopharmaceutical company focused on the development of next-generation immunotherapies for hard-to-treat cancers, today announced the publication of clinical data obtained from the ongoing Phase I study of their rapidly switchable UniCAR platform lead asset, UniCAR-T-CD123, in relapsed/refractory acute myeloid leukemia (rrAML), in the journal “Blood”. The...
Philadelphia, PA – Immunotherapies that target the CD20 antigen have revolutionized how patients with a variety of blood cancers and hematologic disorders have been treated. However, many patients develop resistance to these treatments due to a loss of the antigen that’s being targeted. Now, a new study from researchers at...
Boston, Mass. – A new study co-led by investigators from Mass Eye and Ear, a member of Mass General Brigham, demonstrated the effectiveness of a gene therapy towards restoring hearing function for children suffering from hereditary deafness. In a trial of six children taking place at the Eye & ENT...
LOS ANGELES, Calif. – An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life-threatening immune disorder called severe leukocyte adhesion deficiency-I, or LAD-I, in an international clinical trial co-led by UCLA. LAD-I is a genetic condition that affects approximately one in...
Auckland, New Zealand – A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful...
HONG KONG, BEIJING and BOSTON — GenEditBio Limited (“GenEditBio”), a clinical-stage biotechnology start-up company focusing on genome editing therapeutic solutions through the discovery of novel and precise Cas nucleases and the development of safe and efficient cargo delivery platforms, today announced a key milestone in the development of its pipeline: First patient has been...
GAITHERSBURG, Md. — GeneDx (Nasdaq: WGS), the leader in rare disease diagnosis and improving health through the power of genomic data, today announced a new genetic testing program with Zevra Therapeutics, Inc. (Nasdaq: ZVRA), a commercial-stage biopharmaceutical company focused on bringing life-changing therapeutics to people living with rare diseases. The...