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...
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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...
Sydney, Australia – The epigenetic ‘signature’ of a rare, hard-to-diagnose breast tumour has been found by scientists at the Garvan Institute of Medical Research. The discovery could lead to improved treatment guidelines and better outcomes for patients with this rare disease. Accounting for less than 1% of breast tumours, phyllodes...
REDWOOD CITY, Calif. — Biomea Fusion, Inc. (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing oral covalent small molecules to treat and improve the lives of patients with metabolic diseases and genetically defined cancers, today announced initial response data from the first two type 1 diabetes patients...
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
IRVINE, Calif. – Bioniz Therapeutics, Inc., (“Bioniz”), a clinical-stage biopharmaceutical company advancing a pipeline of first-in-class peptide-based multi-cytokine inhibitors for the treatment of cancer and autoimmune diseases, today announced the European Commission (EC) has granted orphan designation to BNZ-1 for the treatment of cutaneous T-cell lymphoma (CTCL), a rare skin cancer....
MAINZ, Germany and SHANGHAI, China – BioNTech SE (Nasdaq: BNTX) and Duality Biologics (Suzhou) Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for BNT325/DB-1305 for the treatment of patients with platinum-resistant ovarian epithelial cancer, fallopian tube cancer, or primary peritoneal cancer who...
VANCOUVER, BC – BioNxt Solutions Inc. is pleased to report that the comparative pharmacokinetic (PK) study for its oral dissolvable film (ODF) based proprietary Cladribine product for the treatment of Multiple Sclerosis (MS) has been completed. Results will be reported by the Company when available. The PK study closely follows...