[{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not 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Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large 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molecule","year":"2024","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Generium","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Generium \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Generium \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Biogen \/ Not Applicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and Toddlers.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 09, 2024

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Lantesens (nusinersen) is a generic of Spinraza, which is SMN2-directed antisense oligonucleotide investigated for the treatment of spinal muscular atrophy (SMA) in pediatrics.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          September 04, 2024

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Lantesens (nusinersen) is a generic of Spinraza, which is SMN2-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

                          Brand Name : Lantesens

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 14, 2024

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 06, 2024

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 27, 2023

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Biogen

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : $500.0 million

                          January 09, 2023

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Royalty Pharma

                          Deal Size : $1,125.0 million

                          Deal Type : Agreement

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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : $10.0 million

                          January 04, 2023

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Biogen

                          Deal Size : $51.0 million

                          Deal Type : Collaboration

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : RESPOND study is actively enrolling, with baseline characteristics reporting infants and toddlers have residual unmet medical needs in multiple areas after gene therapy; SPINRAZA (nusinersen) treatment following gene therapy was well-tolerated.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 15, 2022

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : The results from NURTURE, a study in infants treated in presymptomatic stage of SMA, demonstrate that early and sustained treatment with SPINRAZA for up to 5.7 years, helped participants to maintain and make progressive gains in motor function.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 14, 2022

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : The ASCEND study aims to evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes in spinal muscular atrophy (SMA) patients.

                          Brand Name : Spinraza

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          September 15, 2021

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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