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NTLA-2002 is the first single-dose treatment being explored for the 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 02, 2024
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