treatment News

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...
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 positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene...
SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company has completed full enrollment in a global Phase 2 randomized, placebo-controlled study of vosoritide, an investigational, once-daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature...
TORONTO, Ontario —  Biomind Labs Inc. (NEO: BMND) (OTC: BMNDF) (FSE: 3XI), a leading biotech company focused on developing the next generation of pharmaceuticals to treat patients suffering from neurological disorders by targeting the drivers of disease, today announced the successful conclusion of the Phase 2 clinical trial of its...
CAMBRIDGE, Mass. and NESS ZIONA, Israel — BiomX Inc., a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that its phage cocktail, BX004, has been granted Orphan Drug Designation (“ODD”) by the United States Food and Drug Administration (“FDA”), for the treatment...
Peer-reviewed publication from Iqbal, et al. describes the multi-site evaluation of optical genome mapping (OGM) for postnatal genetic disorders with 404 samples and shows: Concordance of OGM against various standard of care (SOC) methods –99.5% [399 out of 401 samples] First-pass success rate for OGM – 90.2% [369 out of 409 samples] Overall success rate – 98.8% [404 out of 409 samples] Inter- and intrasite repeatability –100% Pre-print publication from Broeckel, et al. describes the multi-site evaluation of OGM in an additional 560 unique samples, with a total of 749 unique samples to-date from 1,037 datapoints and shows: Concordance for all combined samples against SOC methods – 99.6% [746 out of 749 samples] For a subset of 79 prospectively collected samples from patients suspected of a genetic disorder, SOC had reportable findings in 19 cases [24%]; OGM had reportable findings in 27 cases [34%], corresponding to a 42% increase in the number of cases with reportable findings when OGM was used For another subset of cases consisting of 135 retrospectively collected samples from patients suspected of autism spectrum disorders (ASD), SOC found reportable variants in 63 samples [46%]; OGM identified reportable variants in 83 samples [61%] corresponding to a 32% increase in the number of cases with reportable findings when OGM was used