[{"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Translate Bio Provides MRT5005 Program Updates","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"company":"Dynacure","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","amount":"$54.6 million","upfrontCash":"Undisclosed","newsHeadline":"Dynacure Announces \u20ac50M ($55M) Series C Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Dynacure"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"company":"Vivet Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vivet Therapeutics\u2019 VTX-801 Gets FDA Fast Track Designation for Wilson Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Vivet Therapeutics"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Begins Patient Dosing in US Trial of LYS-GM101 to Treat GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"company":"Coave Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","amount":"$87.3 million","upfrontCash":"$11.6 million","newsHeadline":"Coave Therapeutics and Th\u00e9a Open Innovation Sign Exclusive Licensing, Co-Development and Commercialization Agreement for Europe For CTx-PDE6b, A Novel Gene Therapy Candidate in Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Coave Therapeutics"},{"company":"Dynacure","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Dynacure Announces Strategic Collaboration and Option Agreement with Nippon Shinyaku","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Dynacure"},{"company":"Lysogene","sponsor":"Bpifrance","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lysogene Secures a \u20ac4.3 Million Non-dilutive Financing From Bpifrance to Support Its Development","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UK MHRA Awards Innovation Passport to Lysogene\u2019s Gene Therapy, LYS-GM101 to Treat GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"company":"Dynacure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dynacure Receives Fast Track Designation for DYN101, an Investigational Antisense Oligonucleotide for the Treatment of Myotubular and Centronuclear Myopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Dynacure"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Provides Medical Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"}]
Details:
Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.
Lead Product(s):
MRT5005
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
March 26, 2020
Details:
First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.
Lead Product(s):
MRT5005
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
February 26, 2020
Details:
Lysogene has dosed the first patient in France with LYS-GM101 investigational gene therapy at the Armand Trousseau Hospital (AP-HP). This is the third patient treated with LYS-GM101 in the global adaptative-design clinical trial in children with GM1 gangliosidosis.
Lead Product(s):
AAVrh.10 Expressing Beta-galactosidase
Therapeutic Area: Genetic Disease
Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
February 07, 2022
Details:
Fast Track Designation recieved for DYN101,an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies.
Lead Product(s):
DYN101
Therapeutic Area: Genetic Disease
Product Name: DYN101
Highest Development Status: Phase I/ Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
January 06, 2022
Details:
LYS-GM101 investigational gene therapy an ‘Innovation Passport’ for the treatment of GM1 gangliosidosis under the United Kingdom’s (UK) Innovative Licensing and Access Pathway (ILAP). LYS-GM101 is currently in clinical development.
Lead Product(s):
AAVrh.10 Expressing beta-galactosidase
Therapeutic Area: Genetic Disease
Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
December 20, 2021
Details:
This financing will support the clinical study of LYS-GM101 drug candidate for the treatment of GM1 gangliosidosis and the preclinical studies of LYS-FXS01 in Fragile X syndrome.
Lead Product(s):
LYS-GM101
Therapeutic Area: Genetic Disease
Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Bpifrance
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Financing
November 26, 2021
Details:
Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to exclusively pursue the development and commercialization of DYN101 in Japan depending on analysis of interim data from the Phase 1/2 study, UNITE-CNM.
Lead Product(s):
DYN101
Therapeutic Area: Genetic Disease
Product Name: DYN101
Highest Development Status: Phase I/ Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Nippon Shinyaku
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Agreement
November 10, 2021
Details:
Coave and Théa will co-develop CTx-PDE6b under the joint agreement. Coave retains rights to commercialize CTx-PDE6b in the US and Théa receives an exclusive license to commercialize CTx-PDE6b in Europe (EU27), Ukraine, Turkey, Russia and North Africa.
Lead Product(s):
CTx-PDE6b
Therapeutic Area: Genetic Disease
Product Name: HORA-PDE6b
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Laboratoires Thea
Deal Size: $87.3 million
Upfront Cash: $11.6 million
Deal Type: Licensing Agreement
September 16, 2021
Details:
LYS-GM101 (‘adeno-associated viral vector serotype rh.10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.
Lead Product(s):
AAVrh.10 Expressing Beta-galactosidase
Therapeutic Area: Genetic Disease
Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 31, 2021
Details:
VTX-801 is a novel investigational gene therapy to be evaluated in a Phase 1/2 clinical trial to determine the safety, tolerability, and pharmacological activity of a single intravenous infusion in adult patients with Wilson Disease.
Lead Product(s):
VTX-801
Therapeutic Area: Genetic Disease
Product Name: VTX-801
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Pfizer Inc
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 12, 2021
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