SARASOTA, Fla. and NOVATO, Calif. – GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced that they have received clearance from Health Canada to begin enrolling the Phase 1/2 study of GTX-102 in...
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SHANGHAI, China — GenFleet Therapeutics, a clinical-stage biotechnology company developing cutting-edge therapies in oncology and immunology, announced that U.S. Food and Drug Administration (FDA) has recently granted another two designations for SLS009 (GFH009). GFH009 (highly selective CDK9 inhibitor) so far has received both fast track and orphan drug designations from the...
Genomenon, Inc.®, an AI-driven genomics company, today announced a collaboration with Alexion, AstraZeneca Rare Disease that will make critical information needed for the diagnosis and treatment for a set of rare diseases more readily accessible. The goal of this collaboration is to empower genetic testing labs with the data they need to...
Tokyo, Japan – Researchers in the RIKEN Center for Brain Science (CBS) examined the genetics of autism spectrum disorder (ASD) by analyzing mutations in the genomes of individuals and their families. They discovered that a special kind of genetic mutation works differently from typical mutations in how it contributes to...
DANVILLE, Pa. – Genomic screening to identify hemochromatosis—a disorder that causes iron levels in the body to rise to dangerous levels—encourages people with the condition to seek treatment and ongoing management, a Geisinger study found. Hereditary hemochromatosis type 1 (HH1), caused by a change in the HFE gene, is underdiagnosed, often...
Exclusive rights for novel early-stage asset in cholangiocarcinoma have been acquired by GENFIT in these territories Phase 2 clinical program in cholangiocarcinoma expected to start the first half of 2022 Genoscience Pharma pursues the development with GNS561 in other oncology indications; will start phase 2 in hepatocarcinoma in 2022 Marseille,...
AUSTIN, Texas — Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that in January 2024, the first patient was enrolled and dosed in the Phase 2a expansion portion of the Company’s Acclaim-1 clinical study of Reqorsa® Therapy...
PARIS – GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the independent Data Safety Monitoring Board (DSMB) completed its third safety review of the ongoing PIONEER Phase I/II...
Paris, France – GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that Leber Hereditary Optical Neuropathy (LHON) subjects treated with LUMEVOQ® continued to experience significantly improved vision four...
Genzyme Corporation (NASDAQ: GENZ) today announced that it has detected a virus that impairs cell growth in one of six bioreactors at its Allston Landing manufacturing facility. The company has decided to temporarily interrupt bulk production at the plant to sanitize the facility. Genzyme is collaborating with regulatory agencies as...