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NGN-401 is an investigational AAV9 gene therapy that delivers the full-length human MECP2 gene. It is being evaluated for the treatment of Rett syndrome.
Lead Product(s): NGN-401
Therapeutic Area: Genetic Disease Product Name: NGN-401
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 04, 2024
Details:
The combined company will focus on advancing Neurogene’s pipeline of differentiated genetic medicines, including NGN-401, a clinical-stage product for Rett syndrome, which uses novel gene regulation technology for a potential best-in-class profile.
Lead Product(s): NGN-401
Therapeutic Area: Genetic Disease Product Name: NGN-401
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Recipient: Neoleukin Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Merger December 19, 2023
Details:
NGN-401 is a Cell and Gene therapy drug, administered via intracerebroventricular injection, regulating the MECP2 gene for female pediatric patients with Rett Syndrome.
Lead Product(s): NGN-401
Therapeutic Area: Genetic Disease Product Name: NGN-401
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 30, 2023
Details:
The combined company will focus on advancing Neurogene’s pipeline of differentiated genetic medicines, including NGN-401, a clinical-stage product for Rett syndrome, which uses novel gene regulation technology for a potential best-in-class profile.
Lead Product(s): NGN-401
Therapeutic Area: Genetic Disease Product Name: NGN-401
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Recipient: Neoleukin Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Merger July 18, 2023
Details:
The net proceeds will be used to advance the devlopment of Neurogene's pipeline, including NGN-401, a clinical-stage product for Rett syndrome, which uses novel gene regulation technology for a potential best-in-class profile.
Lead Product(s): NGN-401
Therapeutic Area: Genetic Disease Product Name: NGN-401
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Great Point Partners
Deal Size: $95.0 million Upfront Cash: Undisclosed
Deal Type: Private Placement July 18, 2023
Details:
NGN-401 is an adeno-associated virus (AAV) gene therapy investigational product that is the first to deliver the full-length human MECP2 gene, under the control of Neurogene’s EXACT self-contained gene regulation technology.
Lead Product(s): NGN-401
Therapeutic Area: Genetic Disease Product Name: NGN-401
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 23, 2023
Details:
Proceeds will accelerate clinical development of multiple gene therapy programs, novel gene therapy product designs, next generation technology platform and state-of-the-art AAV Vector GMP manufacturing capabilities.
Lead Product(s): AAV-based gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: EcoR1 Capital
Deal Size: $115.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing December 16, 2020
Details:
The U.S FDA has granted Neurogene the Orphan Drug Designation for its adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease.
Lead Product(s): AAV-based engineered transgene
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 11, 2020
Details:
U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease.
Lead Product(s): Gene Therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 11, 2020
Details:
Dr. Huttner to lead clinical development as Neurogene advances towards first Investigational New Drug (IND) filing.
Lead Product(s): AAV-based gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 15, 2020
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