Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted therapy for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older. The…
A new report titled “Refugee and Migrant Health System Review: Challenges and Opportunities for Long-Term Health System Strengthening in Uganda” was launched on October 15 by Uganda’s Ministry of Health in collaboration with the World Health Organization (WHO). The report…
The pharmaceutical industry is evolving toward sustainability, with increasing awareness of the need for eco-friendly practices. Therapeutic peptides and oligonucleotides (TIDES) present particular challenges due to their traditionally resource-heavy manufacturing processes. With the growing demand for TIDES therapies, the environmental…
Exciting, Challenging, and Insightful – This Is the Impression Pyry Simi, an 18-year-old from Tampere, Gained of the Pharmaceutical Industry After Spending a Day with Outi Vaarala, SVP of Innovative Medicines & R&D at Orion. Through the Job Shadow Day…
AbbVie (NYSE: ABBV) has announced a definitive agreement to acquire Aliada Therapeutics, a biotechnology firm focused on developing therapies using innovative blood-brain barrier (BBB)-crossing technology aimed at treating complex central nervous system (CNS) diseases. Aliada’s lead investigational asset, ALIA-1758, is…
Astellas Pharma Inc. (TSE: 4503; President and CEO: Naoki Okamura) announced today its decision to withdraw the marketing authorization application for avacincaptad pegol intravitreal solution (ACP) from the European Medicines Agency (EMA). ACP is an investigational synthetic aptamer that inhibits…
Judo Bio, a biotechnology company focused on developing oligonucleotide medicines for kidney delivery, has announced the presentation of preclinical data demonstrating a mechanism for the uptake and trafficking of ligand-siRNA conjugates via megalin receptors. This mechanism enables targeted gene knockdown…
Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura) has announced the withdrawal of its marketing authorization application from the European Medicines Agency (EMA) for avacincaptad pegol intravitreal solution (ACP). This investigational synthetic aptamer is designed to inhibit the…
enGene Holdings Inc. (Nasdaq: ENGN), a clinical-stage genetic medicines company, has announced an agreement to sell 6,758,311 of its common shares at a price of $8.90 per share. This financing round is expected to close on October 29, 2024, pending…
Maze Therapeutics, a clinical-stage biopharmaceutical company focused on leveraging human genetics to create innovative small molecule precision medicines for common diseases, has announced promising results from the Phase 1 clinical trial of its drug candidate MZE829 in healthy volunteers. MZE829…
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced updates on its kidney disease pipeline, which includes treatments for IgA nephropathy (IgAN), primary membranous nephropathy (pMN), and APOL1-mediated kidney disease (AMKD). These updates highlight the transformative potential of Vertex’s investigational therapies and include…
GenSight Biologics Reports €3.4 Million Cash Position as of September 30, 2024, and Business Update GenSight Biologics (“GenSight” or the “Company”) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharmaceutical company dedicated to developing innovative gene therapies for retinal neurodegenerative diseases…