Business
Intellia Therapeutics Presents Preclinical Data Demonstrating Advancements in its Broad Genome Editing Capabilities at the 2021 European Society of Gene & Cell Therapy Annual Congress
First preclinical data demonstrating Intellia’s allogeneic platform creates immune-evading T cells for therapeutic use in future cancer treatmentsDemonstrated
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"First preclinical data demonstrating Intellia’s allogeneic platform creates immune-evading T cells for therapeutic use in future cancer treatmentsDemonstrated lipid nanoparticle-based delivery as a more efficient multiplex gene editing approach for engineered cell therapies as compared to electroporation Achieved durable production of normal human alpha-1 antitrypsin protein levels and reduction of endogenous disease-associated protein in non-human primates for the treatment of liver and/or lung manifestations of alpha-1 antitrypsin deficiency (AATD)Platform advances support acceleration of future drug development candidates from both Intellia’s in vivo and ex vivo research portfolio CAMBRIDGE, Mass., Oct. 20, 2021 (GLOBE NEWSWIRE) -- 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 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. The data shared showed that Intellia’s allogeneic platform, leveraging a novel combination of sequential gene edits, can prevent immune rejection of allogeneic T cells in in vitro and in vivo models for future application in TCR-T and CAR-T therapy. Additionally, data highlighted that lipid nanoparticles (LNPs) can replace electroporation for delivery of CRISPR/Cas9 gene edits to T cells, avoiding the risk of chromosomal translocations observed when multiple edits are performed simultaneously, as well as the negative effect of electroporation on T cell health. Finally, results from an ongoing study demonstrated proof-of-concept in non-human primates (NHPs) for in vivo gene insertion and knockout for the treatment of alpha-1 antitrypsin deficiency (AATD), which resulted in sustained production of normal human levels of healthy alpha-1 antitrypsin (A1AT) protein and reduction of the endogenous disease-associated protein. The data were presented at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) meeting, taking place virtually from October 19 – 22, 2021. “Preclinical data presented at ESGCT’s Annual Congress show that using our proprietary genome editing platform, Intellia is able to accomplish mul...