LEXINGTON, Mass. — Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced new data from its two preclinical programs targeting pathological tau for the treatment of Alzheimer’s disease. Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy program...
treatment News
Cambridge, England – Voydeya (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular haemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris...
WILMINGTON, Del. — AstraZeneca Pharmaceuticals LP and Ionis’ WAINUA™ (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered...
CAMBRIDGE, Mass. — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive data from the Phase 2 FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne...
WASHINGTON DC – Gene-editing technologies are progressing fast, and there are now more than 100 ongoing trials for new therapies and diagnostics based on CRISPR-Cas, zinc finger nucleases, TALENS, and base editors. While most of these trials are evaluating ex vivo gene-edited cell-based therapies for cancers and genetic blood disorders,...
Guildford, England. Insulin icodec, a once-weekly basal injection to treat type 1 diabetes, has the potential to be as effective in managing the condition as daily basal insulin treatments, according to research from the University of Surrey. The results of the year-long phase 3 clinical trial could revolutionise the future...
New York, NY – Patients who have drug-resistant tuberculosis (TB) have a similar microbiological response to bedaquiline-based second-line medications as patients with drug-sensitive TB taking first-line regimens, according to researchers at Weill Cornell Medicine in New York and GHESKIO Centers in Haiti. Second-line medications are those that are given when...
Cambridge, UK – A new, systematic analysis of cancer cells identifies 370 candidate priority drug targets across 27 cancer types, including breast, lung and ovarian cancers. By looking at multiple layers of functional and genomic information, researchers were able to create an unbiased, panoramic view of what enables cancer cells...
CHENGDU, China — Kicking off the New Year with remarkable achievements, Chengdu-based WestGene made a significant impact at the 3rd mRNA-Based Therapeutics Summit in Berlin, Germany. Renowned for her pivotal role in mRNA research, Dr. Xiangrong Song, co-founder and CEO of WestGene, presented the latest advances in the company’s oncology...
Gene-targeted therapies have the potential to revolutionize medicine through treatments that modify or replace disease-causing genes with healthy variants. Gene therapy commonly involves using a modified virus to deliver the healthy genes into a patient’s cells. Once there, the new genetic material instructs the cell to produce the missing or...