CAMBRIDGE, Mass. – Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced that the U.S. Food and Drug Administration (FDA) has accepted the investigational new drug (IND) application for NTLA-5001, the company’s first wholly-owned ex vivoCRISPR genome...
treatment News
CAMBRIDGE, Mass. – Intellia Therapeutics, Inc.(NASDAQ:NTLA) today announced that a late-breaking abstract featuring interim Phase 1 clinical data from NTLA-2001, the Company’s lead CRISPR/Cas9 in vivo therapy in development as a single-dose, systemically administered treatment for transthyretin (ATTR) amyloidosis, has been selected for an oral presentation at the 2021 Peripheral...
CAMBRIDGE, Mass. — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that longer-term data from the ongoing Phase 1 trial of investigational nex-z for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) will be presented at the 5th International...
MORRISTOWN, N.J. — Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA)...
Sendai, Japan – An interim analysis of an ongoing Japanese study published in Modern Rheumatology demonstrates that mepolizumab is safe and effective in improving disease control among patients with eosinophilic granulomatosis with polyangiitis (EGPA), a form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). Remission is typically achieved and maintained in...
Boston, MA – A new international consensus report on precision medicine in diabetes prevention and care highlights the significant advancements in precision medicine in diabetes prevention, diagnosis, treatment, and prognosis while also shedding light on numerous knowledge gaps. The report, Second international consensus report on gaps and opportunities for the...
Barcelona, Spain – An international team of scientists led by ICREA researcher and Director of the Life Sciences Department at the Barcelona Supercomputing Centre – Centro Nacional de Supercomputación (BSC-CNS), Alfonso Valencia, has developed a technology based on artificial intelligence (AI) for the study of orphan diseases and has successfully...
Nashville, Tennessee – Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD). The therapy, nonmyeloablative haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy), is proving to have equivalent efficacy...
AUSTIN, TX – IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients...
Daix (France) and New York, NY – Inventiva (Euronext Paris and Nasdaq: IVA) (“Inventiva” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of oral therapies for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), today announces the publication in Journal of Hepatology Reports, a peer-reviewed, scientific journal, of results...