Intellia Therapeutics Inc (NTLA): * INTELLIA THERAPEUTICS RECEIVES U.S. FDA ORPHAN DRUG DESIGNATION FOR NTLA-2001, AN INVESTIGATIONAL CRISPR THERAPY FOR THE TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS Source text for Eikon: Further company coverage:
Intellia Therapeutics, Inc. (NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced today that the U.S. Food and Drug Administration has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin amyloidosis.
Intellia Therapeutics Inc (NTLA): * INTELLIA THERAPEUTICS PRESENTS PRECLINICAL DATA DEMONSTRATING ADVANCEMENTS IN ITS BROAD GENOME EDITING CAPABILITIES AT THE 2021 EUROPEAN SOCIETY OF GENE & CELL THERAPY ANNUAL CONGRESS Source text for Eikon: Further company coverage:
CAMBRIDGE, Mass., Oct. 20, 2021 -- Intellia Therapeutics, Inc. (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 new data supporting novel capabilities of its CRISPR/Cas9 genome editing platform, which the Company plans to leverage for the development of future therapeutic candidates.
Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. It utilizes a biological tool known as the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. The…
Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. It utilizes a biological tool known as the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 for the treatment of hereditary angioedema, as well as other liver-focused programs comprising hemophilia A and hemophilia B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo pipeline includes NTLA-5001 for the treatment of acute myeloid leukemia; and proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune disorders. Intellia Therapeutics, Inc. has license and collaboration agreements with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for the treatment of sickle cell disease; Regeneron Pharmaceuticals, Inc. to co-develop potential products for the treatment of hemophilia A and hemophilia B; and Ospedale San Raffaele. The company was formerly known as AZRN, Inc. and changed its name to Intellia Therapeutics, Inc. in July 2014. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.
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