Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Neurogene Reports Positive Data for NGN-401 in Rett Syndrome Trial
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
November 11, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Neurogene Receives RMAT Designation For Rett Syndrome Gene Therapy NGN-401
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 07, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Neurogene Announces NGN-401 Gene Therapy for Rett Syndrome Selected by FDA Pilot Program
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 03, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Neurogene Expands and Plans Rapid Patient Enrollment for Rett Syndrome Gene Therapy Trial
Details : 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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
March 04, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Recipient : Neoleukin Therapeutics
Deal Size : Undisclosed
Deal Type : Merger
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
December 19, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Recipient : Neoleukin Therapeutics
Deal Size : Undisclosed
Deal Type : Merger
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
November 30, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Recipient : Neoleukin Therapeutics
Deal Size : Undisclosed
Deal Type : Merger
Neurogene and Neoleukin Announce Definitive Merger Agreement
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
July 18, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Recipient : Neoleukin Therapeutics
Deal Size : Undisclosed
Deal Type : Merger
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Great Point Partners
Deal Size : $95.0 million
Deal Type : Private Placement
Neurogene and Neoleukin Announce Definitive Merger Agreement
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
July 18, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Great Point Partners
Deal Size : $95.0 million
Deal Type : Private Placement
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome
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.
Brand Name : NGN-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
January 23, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : AAV-based gene therapy
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : EcoR1 Capital
Deal Size : $115.0 million
Deal Type : Series B Financing
Neurogene Closes $115 Million Series B Financing
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.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
December 16, 2020
Lead Product(s) : AAV-based gene therapy
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : EcoR1 Capital
Deal Size : $115.0 million
Deal Type : Series B Financing
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