treatment News

CAMBRIDGE, Mass. and TARRYTOWN, N.Y. – Intellia Therapeutics, Inc.(NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive interim data from an ongoing Phase 1 clinical study of their lead in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. The Phase 1...
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 interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM). NTLA-2002 is...
CAMBRIDGE, Mass. — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today presented additional interim results from its ongoing Phase 1 study of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for...
CAMBRIDGE, Mass. – Intellia Therapeutics, Inc. (NASDAQ:NTLA) announced today that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has approved a protocol amendment for the Company’s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study of NTLA-2001 now includes patients...
Cambridge, Mass. — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE), enabling...
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...
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...
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...