
													
															
															Year
															DEALS // DEV.
															Country
															
															Therapeutic Area
															Study Phase
															Deal Type
															Product Type
															Dosage Form
															Lead Product
															Target

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
November 11, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
August 07, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
June 03, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
March 04, 2024
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
December 19, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Recipient : Neoleukin Therapeutics
Deal Size : Undisclosed
Deal Type : Merger

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
November 30, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
July 18, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Great Point Partners
Deal Size : $95.0 million
Deal Type : Private Placement

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
July 18, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Recipient : Neoleukin Therapeutics
Deal Size : Undisclosed
Deal Type : Merger

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Details : NGN-401 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Rett Syndrome.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
June 12, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
January 23, 2023
Lead Product(s) : NGN-401
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
