Philadelphia, PA – Millions of people living with neurodegenerative diseases and their loved ones have long hoped for the day when treatments could change the prognosis from an inevitable decline to a managed, stable condition. The urgent need for life-changing therapies exists against the backdrop of a growing prevalence of...
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SAN DIEGO, Calif. — Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer therapeutics, presented promising preclinical data in two programs, OPN-6602, a dual EP300/CBP inhibitor in multiple myeloma, and OPN-9840, an oral, non-covalent TEAD inhibitor in malignant mesothelioma and metastatic melanoma. Data were...
SINGAPORE – Keeping blood pressure in check could save lives among Asian adults with type 2 diabetes, suggests a sizable new study of more than 80,000 patients in Singapore. But how low should it go? A new study led by Duke-NUS Medical School—published in the Journal of the American Heart...
BEIJING & CAMBRIDGE, Mass. – CANbridge Pharmaceuticals, Inc., a biopharmaceutical company developing and commercializing innovative drugs to treat rare diseases and rare cancers in China and globally, announced that it has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company...
Selinexor once weekly can be safely combined with pomalidomide and dexamethasone in heavily pretreated patients with multiple myeloma (MM), according to the results of a study presented at the 2021 American Society of Clinical Oncology Annual Meeting. In the multi-arm phase 1b/2 STOMP study (ClinicalTrials.gov Identifier: NCT02343042), investigators evaluated selinexor at...
BOSTON, LONDON and LEIDEN, The Netherlands – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global, commercial stage biopharmaceutical company, today announced a strategic collaboration to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivo autologous hematopoietic...
BOSTON and LONDON – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) evaluating the safety and efficacy of OTL-203 for the treatment of the Hurler subtype of Mucopolysaccharidosis type I (MPS-IH). OTL-203 is an investigational autologous hematopoietic stem cell...
BOSTON and LONDON – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the appointment of two gene therapy and industry experts, Fulvio Mavilio, Ph.D., and Nicoletta Loggia, Ph.D., to serve as chief scientific officer and chief technical officer, respectively. The company also announced the promotion of Leslie Meltzer, Ph.D.,...
TOKYO, LONDON and BOSTON — Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, for the treatment of children...
BOSTON and LONDON — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I...