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Details:
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.
Lead Product(s): CRISPR-based Therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Regeneron Pharmaceuticals
Deal Size: $420.0 million Upfront Cash: $100.0 million
Deal Type: Termination March 22, 2024
Details:
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.
Lead Product(s): NTLA-2001
Therapeutic Area: Genetic Disease Product Name: NTLA-2001
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 18, 2024
Details:
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.
Lead Product(s): CRISPR-based Therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery PlatformProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: ReCode Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration February 15, 2024
Details:
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).
Lead Product(s): NTLA-2002
Therapeutic Area: Genetic Disease Product Name: NTLA-2002
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 14, 2023
Details:
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.
Lead Product(s): NTLA-2001
Therapeutic Area: Genetic Disease Product Name: NTLA-2001
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 18, 2023
Details:
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).
Lead Product(s): NTLA-2002
Therapeutic Area: Genetic Disease Product Name: NTLA-2002
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 13, 2023
Details:
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
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: Discovery PlatformProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Regeneron Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Expanded Collaboration October 03, 2023
Details:
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.
Lead Product(s): NTLA-2002
Therapeutic Area: Genetic Disease Product Name: NTLA-2002
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 11, 2023
Details:
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).
Lead Product(s): NTLA-2002
Therapeutic Area: Genetic Disease Product Name: NTLA-2002
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 21, 2023
Details:
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).
Lead Product(s): NTLA-2002
Therapeutic Area: Genetic Disease Product Name: NTLA-2002
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 02, 2023
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