RESEARCH TRIANGLE PARK, N.C. — Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that its investigational LCA5 gene therapy program, OPGx-LCA5, has been accepted into the U.S. Food...
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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...
PALO ALTO, Calif. — Orbus Therapeutics, Inc., a private pharmaceutical company, today announced the publication of the Phase 3 STELLAR clinical study results demonstrating that the combination of eflornithine and lomustine achieved clinically meaningful improvements in OS and PFS compared to lomustine alone in patients with recurrent IDH mutant, grade...
PALO ALTO, Calif. — Orbus Therapeutics, Inc., a private pharmaceutical company, today announced the presentation of the Phase 3 STELLAR clinical study results showing clinically meaningful benefits in patients with grade 3 astrocytoma—an aggressive brain tumor with a median survival of only two to five years post-diagnosis. The results will...
MENLO PARK, Calif. — Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) seeking approval for Orca-T, its lead investigational allogeneic T-cell immunotherapy,...
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...
LONDON, UK — Orchard Therapeutics, a Kyowa Kirin company, announced it has been granted Innovation Passport designation for OTL-201, an investigational haemopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A). The designation was awarded under the...
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, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The...