CAMBRIDGE, Mass. – Voyager Therapeutics, Inc. (Nasdaq: VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe neurological diseases, today announced the U.S. Food and Drug Administration (FDA) has removed its clinical hold on the company’s Investigational New Drug (IND) application for VY-HTT01, a gene therapy candidate for the...
treatment News
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...
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 it has regained full rights to WVE-006, an investigational GalNAc-conjugated RNA editing oligonucleotide (AIMer) for alpha-1 antitrypsin deficiency (AATD), from GSK. This...
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 200 mg single and multidose and 400 mg single dose cohorts of the ongoing Phase 1b/2a RestorAATion-2 study...
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,...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced topline results from Week 52 of COACH, the first Phase 2 clinical trial to evaluate combination therapy with once-weekly TransCon CNP (navepegritide) and once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia. At Week 52, combination therapy showed durable growth without...
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...