PARIS, France – The time from symptom onset to receiving a diagnosis of Gaucher disease has significantly decreased over the last decades, from a median of 5.4 years before 2000 to less than one year after 2020, a study in France has found. Still, researchers noted that, while most patients received...
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WATERTOWN, Mass. — Diagonal Therapeutics, a biotechnology company focused on correcting dysregulated signaling with clustering antibodies that address the underlying cause of intractable genetic diseases, today announced the publication of study results detailing new health economics and outcomes research (HEOR) that characterizes the significant healthcare utilization and costs associated with hereditary...
DIAG723 is supported by a groundbreaking preclinical profile demonstrating its disease-modifying potential WATERTOWN, Mass. — Diagonal Therapeutics, a biotechnology company focused on correcting dysregulated signaling with clustering antibodies that address the underlying cause of intractable genetic diseases, today announced that both the U.S. Food and Drug Administration (FDA) granted orphan drug...
HOUSTON, TX — Diakonos Oncology Corporation, a clinical stage immuno-oncology company, announced today it has completed enrollment for its Phase 1 trial of DOC1021, a unique dendritic cell vaccine, for Glioblastoma Multiforme (GBM). With the first patient enrolled in October 2021, DOC1021 has been administered to 16 patients across four...
HOUSTON, TX – The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Diakonos’ dendritic cell vaccine DOC1021 for malignant glioma. If the therapy is approved, the US-based immuno-oncology company is eligible for a potential seven years of market exclusivity in newly diagnosed or refractory glioblastoma multiforme,...
NEW YORK, NY and WALTHAM, Mass. – Dianthus Therapeutics, Inc. (Nasdaq: DNTH), a clinical-stage biotechnology company dedicated to advancing the next generation of antibody complement therapeutics to treat severe autoimmune diseases, today announced the initiation of the Phase 2 MaGic trial of DNTH103 in patients with generalized Myasthenia Gravis. The...
LEXINGTON, Mass. – Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced interim results from the four completed active-treatment dose cohorts (0.1, 1.0, 3.0 and 6.0 mg/kg) of its Phase 1 double-blind, placebo-controlled, randomized trial of belcesiran, an investigational GalXC™ RNAi therapeutic...
LEXINGTON, Mass. – Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, announced the appointment of Rob Ciappenelli as Chief Strategy Officer, the promotion of Marc Abrams, Ph.D., to Senior Vice President, Discovery Research and the addition of Kristen Sheppard,...
LEXINGTON, Mass. – Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced top-line results from its PHYOX4 study designed to evaluate the safety and tolerability of a single subcutaneous dose of nedosiran, Dicerna’s late-stage investigational GalXC™ RNAi therapeutic candidate in development for...
LEXINGTON, Mass. – Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, announced it has initiated patient dosing in the Company’s Phase 2 ESTRELLA trial of belcesiran, an investigational GalXC™ RNAi therapeutic candidate for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver...