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    [{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Pharming","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Recombinant","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pharming \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Morningside Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Series A Financing","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Morningside Ventures","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Morningside Ventures"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Sands Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Series B Financing","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Sands Capital","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Sands Capital"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Ferrer Internacional","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Licensing Agreement","leadProduct":"Pridopidine","moa":"Dopamine D2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Prilenia therapeutics","amount2":0.56999999999999995,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56999999999999995,"dosageForm":"Capsule","sponsorNew":"Prilenia therapeutics \/ Grupo Ferrer Internacional","highestDevelopmentStatusID":"10","companyTruncated":"Prilenia therapeutics \/ Grupo Ferrer Internacional"},{"orgOrder":0,"company":"Khondrion","sponsor":"Netherlands Enterprise Agency","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Funding","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Khondrion","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Solution","sponsorNew":"Khondrion \/ Netherlands Enterprise Agency","highestDevelopmentStatusID":"10","companyTruncated":"Khondrion \/ Netherlands Enterprise Agency"},{"orgOrder":0,"company":"Khondrion","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Khondrion \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"UMDF","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2024","type":"Funding","leadProduct":"Sonlicromanol","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ UMDF","highestDevelopmentStatusID":"10","companyTruncated":"Khondrion \/ UMDF"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Khondrion

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                            Khondrion

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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Netherlands Enterprise Agency

                            Deal Size : $5.6 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Khondrion gets $5M Credit Plus more Funds for Mitochondrial Trial

                            Details : The funding aims to advance the clinical development of Khondrion lead product product KH176 (sonlicromanol), which is being evaluated for the treatment of Primary mitochondrial Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            May 06, 2025

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Netherlands Enterprise Agency

                            Deal Size : $5.6 million

                            Deal Type : Funding

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                            02

                            Prilenia therapeutics

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                            ISPE Annual Meeting
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                            Prilenia therapeutics

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                            • fda
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                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Ferrer Internacional

                            Deal Size : $568.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Ferrer and Prilenia Partner for Pridopidine Commercialization and Co-Development

                            Details : Under the licensing agreement, Ferrer will holds the right to develop TV-7820 (pridopidine) an orally administered sigma-1 receptor agonist. It is being evaluated for Huntington’s Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : $90.8 million

                            April 28, 2025

                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Ferrer Internacional

                            Deal Size : $568.0 million

                            Deal Type : Licensing Agreement

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                            03

                            Khondrion

                            Netherlands arrow
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                            Khondrion

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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : UMDF

                            Deal Size : Undisclosed

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            UMDF Supports Khondrion Drug Development for Primary Mitochondrial Disease

                            Details : The proceeds will be used for the development of KH176 (sonlicromanol), a first-in-class, brain-penetrant redox-modulator, for adults with primary mitochondrial disease due to the m.3243A>G mutation.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            November 21, 2024

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : UMDF

                            Deal Size : Undisclosed

                            Deal Type : Funding

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                            04

                            Khondrion

                            Netherlands arrow
                            ISPE Annual Meeting
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                            Khondrion

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                            ISPE Annual Meeting
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                            • fda
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                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Khondrion Gets FDA Clearance for Sonlicromanol Phase 3 in Mitochondrial Disease

                            Details : KH176 (sonlicromanol) is a mPGES-1 inhibitor small molecule drug candidate, which is being evaluated for the treatment of adult patients with PMD due to the m.3243A>G mutation.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 14, 2024

                            Lead Product(s) : Sonlicromanol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Prilenia therapeutics

                            Netherlands arrow
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                            Prilenia therapeutics

                            Netherlands arrow
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                            • fda
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                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Prilenia Achieves Last Patient Last Visit in Phase 3 PROOF-HD Clinical Study for Huntington’s Disease

                            Details : Pridopidine is an oral, highly selective S1R agonist which stimulates multiple cellular pathways, including autophagy, which are essential to neuronal function and survival, and may lead to neuroprotective effects.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 28, 2023

                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Uniqure

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                            Uniqure

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                            Lead Product(s) : Etranacogene Dezaparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : CSL Behring

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvove...

                            Details : Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable FIX expression, assessed at 18 months following a single dose of AMT-061(etranacogene dezaparvovec).

                            Product Name : Hemgenix

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            December 09, 2021

                            Lead Product(s) : Etranacogene Dezaparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : CSL Behring

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Prilenia therapeutics

                            Netherlands arrow
                            ISPE Annual Meeting
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                            Prilenia therapeutics

                            Netherlands arrow
                            ISPE Annual Meeting
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                            • fda
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                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Prilenia Receives Fast Track Designation for Pridopidine for the Treatment of Huntington’s Disease

                            Details : Fast Track accelerate the registration process for pridopidine by providing the ability to file a rolling NDA and qualify for priority review, and administered orally acts as a highly selective and potent Sigma-1 Receptor agonist is for HD and ALS.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 17, 2021

                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Prilenia therapeutics

                            Netherlands arrow
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                            Not Confirmed

                            Prilenia therapeutics

                            Netherlands arrow
                            ISPE Annual Meeting
                            Not Confirmed
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                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Sands Capital

                            Deal Size : $43.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Prilenia Closes Oversubscribed $43 Million in Series B Financing Round

                            Details : The proceeds will be used to prepare for potential registration and commercialization of its lead drug candidate, pridopidine, for patients with Huntington’s Disease (HD) and Amyotrophic Lateral Sclerosis (ALS).

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            November 03, 2021

                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Sands Capital

                            Deal Size : $43.0 million

                            Deal Type : Series B Financing

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                            09

                            Prilenia therapeutics

                            Netherlands arrow
                            ISPE Annual Meeting
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                            Prilenia therapeutics

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                            ISPE Annual Meeting
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                            • fda
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                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Prilenia’s Phase 3 Huntington’s Disease Clinical Trial Achieves 50% Enrollment

                            Details : The trial is investigating Prilenia’s lead candidate, pridopidine, for treatment of Huntington’s Disease. The study reached 25% in May 2021 and enrolled a further 25% in only six weeks, with now over 240 registered early-stage HD patients.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 08, 2021

                            Lead Product(s) : Pridopidine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Uniqure

                            Netherlands arrow
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                            Uniqure

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                            Lead Product(s) : Etranacogene Dezaparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : CSL Behring

                            Deal Size : $2,050.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            UniQure Announces Closing of Commercialization and License Agreement With CSL Behring

                            Details : uniQure announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec expired on May 5, 2021, and the agreement became fully effective on...

                            Product Name : Hemgenix

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : $450.0 million

                            May 06, 2021

                            Lead Product(s) : Etranacogene Dezaparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : CSL Behring

                            Deal Size : $2,050.0 million

                            Deal Type : Licensing Agreement

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