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...
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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...
BOSTON and LONDON — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, announced various business accomplishments along with its financial results for the quarter ended March 31, 2023. “Following multiple productive interactions this year with the FDA on our clinical and CMC packages, I am delighted to announce the...
Boston and London — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, announced that the company will make a virtual presentation at the Guggenheim Genomic Medicines and Rare Disease Day on Tuesday, April 4, 2023 at 1:35pm ET. A live webcast of the presentation will be available under “News...
PORTLAND, Oregon — An artificial intelligence technology can accurately and independently detect 100% of severe cases of a blindness-causing condition that affects prematurely born babies, according to new research from Oregon Health & Science University and collaborators that was published today in the journal JAMA Ophthalmology. The technology has the potential...
CORVALLIS, Ore. – A study spearheaded by Oregon State University has shown why certain polyunsaturated fatty acids work to combat a dangerous liver condition, opening a new avenue of drug research for a disease that currently has no FDA-approved medications. Scientists led by Oregon State’s Natalia Shulzhenko, Andrey Morgun and...