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Find Clinical Drug Pipeline Developments & Deals by Intellia Therapeutics
Through the termination, Intellia opted out of a partnership with Regeneron to develop a factor IX gene editing therapy, for the treatment of hemophilia A and B.
NTLA-2001 is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. It is being evaluated for the treatment of transthyretin amyloidosis with cardiomyopathy.
The collaboration combines Intellia’s leading CRISPR-based platform, including its DNA writing technology, with ReCode’s proprietary selective Organ Targeting lipid nanoparticle to precisely correct one or more cystic fibrosis disease-causing gene mutations.
NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE).
NTLA-2001 is the first-ever investigational in vivo CRISPR-based gene editing therapy, which is investigated for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.
NTLA-2002, an investigational CRISPR therapeutic candidate, is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE).
The collaboration will leverage Regeneron’s proprietary antibody-targeted AAV vectors and delivery systems and Intellia’s proprietary Nme2 CRISPR/Cas9 systems to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases.
Lead Product(s):
CRISPR-based Gene Editing Therapy
NTLA-2002 is a investigational CRISPR therapeutic candidate designed to inactivate the KLKB1 gene and potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema.
NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE).
NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. It is being developed for hereditary angioedema (HAE).