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Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Prevail Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"PR006","moa":"Progranulin","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Prevail Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Prevail 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Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Prevail Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"PR006","moa":"Progranulin","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Prevail Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Prevail Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Prevail Therapeutics \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Acquisition will set up a gene therapy program at Lilly, based on Prevail's portfolio of neuroscience assets, including the lead candidate, PR001, and will broaden Lilly's commitment to use novel modalities to address fatal genetic forms of neurodegenera...

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 22, 2021

                          Lead Product(s) : PR001,Methylprednisolone

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Eli Lilly

                          Deal Size : $1,040.0 million

                          Deal Type : Acquisition

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                          02

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Acquisition will establish a gene therapy program at Lilly, anchored by Prevail's portfolio of neuroscience assets, and will broaden Lilly's commitment to use novel modalities to attempt to address otherwise fatal genetic forms of neurodegenerative disea...

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 15, 2020

                          Lead Product(s) : PR001,Methylprednisolone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Eli Lilly

                          Deal Size : $1,040.0 million

                          Deal Type : Acquisition

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                          03

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The Company also announced that the FDA has granted Rare Pediatric Disease Designation for PR001 for the treatment of neuronopathic Gaucher disease (nGD), the most severe form of the condition.

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 02, 2020

                          Lead Product(s) : PR001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : PR006 is an investigational AAV9 gene therapy delivering the GRN gene and is being developed as a potential therapy for patients with frontotemporal dementia with GRN mutations.

                          Product Name : PR006

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 30, 2020

                          Lead Product(s) : PR006,Methylprednisolone

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for...

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 18, 2020

                          Lead Product(s) : PR001,Methylprednisolone

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The Company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). Prevail expects to start enrollment of the PROVIDE Phase 1/2 clinical trial of PR001 in the second half of 2020.

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          October 27, 2020

                          Lead Product(s) : PR001,Methylprednisolone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Company Advancing its Phase 1/2 Clinical Trial for the Treatment of Neuronopathic Gaucher Disease and Expects to Initiate Patient Dosing in First Half of 2020.

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 01, 2020

                          Lead Product(s) : PR001

                          Therapeutic Area : Oncology

                          Highest Development Status : IND Enabling

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in the GRN gene that reduce production of progranulin.

                          Product Name : PR006

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          July 27, 2020

                          Lead Product(s) : PR006

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : Preclinical data demonstrate potential of Prevail’s AAV gene therapy approach to slow or halt progression in multiple neurodegenerative diseases.

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 29, 2020

                          Lead Product(s) : PR001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Peptide Therapy Summit
                          Not Confirmed
                          Peptide Therapy Summit
                          Not Confirmed

                          Details : The presentation will highlight preclinical data related to PR001 for PD-GBA and the design of PROPEL, Phase 1/2 clinical trial to evaluate the safety and efficacy of PR001 in patients with PD-GBA.

                          Product Name : PR001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 31, 2020

                          Lead Product(s) : PR001

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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