Salanersen (BIIB115/ION306) is a novel antisense oligonucleotide (ASO) with the potential to achieve high efficacy and once yearly dosing in spinal muscular atrophy (SMA) Interim Phase 1 data show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function...
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NEW HAVEN, Conn. – Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), announced the enrollment of the first patient in a Phase 1a/1b trial in Multiple Myeloma using the ARM, BHV-1100, in combination with autologous cytokine induced memory-like (CIML) natural killer (NK) cells and immunoglobulin (Ig) to target and kill multiple...
NEW HAVEN, Conn. and LEUVEN, Belgium – Biohaven Therapeutics Ltd., a subsidiary of Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), along with CD3 and LICR at KU Leuven, announced that they have entered into an exclusive global license and research agreement to develop and commercialize first-in-class TRPM3 antagonists to address...
LUND, Sweden – BioInvent International AB (“BioInvent”)(Nasdaq Stockholm: BINV)(STO:BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, today announced that the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) has adopted a positive opinion for Orphan...
TEL AVIV, Israel — BioLineRx Ltd., a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced that the first patient has been dosed in the l evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies...
TEL AVIV, Israel – BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, today announced positive top-line results from the Company’s GENESIS Phase 3 trial evaluating its lead clinical candidate, Motixafortide, in combination with granulocyte colony stimulating factor (G-CSF, the standard of care in this...
SARASOTA, Fla. – Biom Therapeutics, a clinical stage biotech company, announced today it has received orphan drug designation status for BIO017—which is designed to treat Angelman syndrome—from the U.S. FDA’s Office of Orphan Product Development. Biom anticipates commencing clinical trials for BIO017 in Q4 of 2021, following submission of an...
RMAT Designation Granted by FDA During Bleeding Disorders Awareness Month SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A....
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone. Safety results were consistent with the...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data characterizing the efficacy and safety of PALYNZIQ® (pegvaliase-pqpz) for the treatment of adolescents with phenylketonuria (PKU), which were presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, Sept. 2-6, 2025. The Phase 3 PEGASUS study...