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AB-1002 (NAN-101) is an investigational gene therapy, which is currently under phase 2 clinical development for the treatment of congestive heart failure.
Lead Product(s): NAN-101
Therapeutic Area: Cardiology/Vascular Diseases Product Name: AB-1002
Highest Development Status: Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Bayer AG
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 16, 2024
Details:
AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. It is under phase 1 clinical development for the treatment of Parkinson's disease.
Lead Product(s): AAV2-GDNF
Therapeutic Area: Rare Diseases and Disorders Product Name: AB-1005
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Bayer AG
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 04, 2024
Details:
AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. It is under phase 1 clinical development for the treatment of Parkinson's disease.
Lead Product(s): AB-1005
Therapeutic Area: Rare Diseases and Disorders Product Name: AB-1005
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 November 17, 2023
Details:
AB-1003 (also known as LION-101) is a novel investigational recombinant adeno-associated virus based gene therapy currently being developed as a one-time intravenous infusion for the treatment of patients with LGMD type 2I/R9.
Lead Product(s): AB-1003
Therapeutic Area: Genetic Disease Product Name: AB-1003
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 August 03, 2023
Details:
AB-1005 is an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of mild to moderate parkinson’s disease.
Lead Product(s): AB-1005
Therapeutic Area: Neurology Product Name: AB-1005
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 March 29, 2023
Details:
AB-1003 is a novel investigational recombinant adeno-associated virus based gene therapy currently being developed as a one-time intravenous infusion for the treatment of patients with LGMD type 2I/R9.
Lead Product(s): AB-1003
Therapeutic Area: Genetic Disease Product Name: AB-1003
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 February 27, 2023
Details:
AskBio will combine its synthetic DNA and gene editing nucleases with ReCode's selective organ targeting LNP technology to potentially create an all-in-one solution that enables full gene insertion by delivering with precision both the gene editing tool and DNA.
Lead Product(s): Undisclosed
Therapeutic Area: Technology 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 January 09, 2023
Details:
ACT-101 (also known as ACTUS-101) is an investigational gene therapy developed by Actus Therapeutics, a member of AskBio, for the treatment of Pompe disease. It targets the underlying cause of the disorder by using a modified, harmless adeno-associated virus (AAV).
Lead Product(s): ACTUS-101
Therapeutic Area: Genetic Disease Product Name: ACTUS-101
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 December 20, 2022
Details:
BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein.
Lead Product(s): BV-101
Therapeutic Area: Genetic Disease Product Name: BV-101
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Bayer AG
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 23, 2022
Details:
NAN-101 is an adeno-associated virus (AAV) gene therapy designed to stimulate protein phosphatase inhibitor 1 (I-1c) to block the action of protein phosphatase 1 (PP1), which is linked to heart failure.
Lead Product(s): NAN-101
Therapeutic Area: Cardiology/Vascular Diseases Product Name: NAN-101
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Bayer AG
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 16, 2022
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