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    [{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Agreement","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":1.1299999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1299999999999999,"dosageForm":"Intrathecal Injection","sponsorNew":"Ionis Pharmaceuticals \/ Royalty Pharma","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Royalty Pharma"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"15","companyTruncated":"Alcyone Therapeutics \/ Biogen"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Collaboration","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Alcyone Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Intrathecal Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"15","companyTruncated":"Alcyone Therapeutics \/ Biogen"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Generium","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"RUSSIA","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Generium","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Generium \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Generium \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"}]

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                            01

                            Biogen

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                            Biogen

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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            January 23, 2025

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Biogen

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                            Peptide Process CMC
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                            Biogen

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                            Peptide Process CMC
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biogen Reports Promising Results For Higher Dose of Spinraza

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            September 10, 2024

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Biogen

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                            Peptide Process CMC
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                            Biogen

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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRxâ„¢ System...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            August 15, 2024

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Biogen

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                            Peptide Process CMC
                            Not Confirmed

                            Biogen

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                            Peptide Process CMC
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Biomarker Data Support Potential Benefit of SPINRAZA in Infants

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            June 03, 2024

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Generium

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                            Peptide Process CMC
                            Not Confirmed

                            Generium

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                            Peptide Process CMC
                            Not Confirmed
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Russia registers domestic generic of Spinraza

                            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.

                            Product Name : Lantesens

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            May 14, 2024

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Biogen

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                            Peptide Process CMC
                            Not Confirmed

                            Biogen

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                            Peptide Process CMC
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biogen Reports Positive Topline Results For Higher Dose Of Nusinersen in SMA

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 09, 2024

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Royalty Pharma

                            Deal Size : $1,125.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Ionis and Royalty Pharma Enter Into Royalty Agreement for Up to $1.1 Billion to Further Advance Ionis' Genetic...

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : $500.0 million

                            September 01, 2023

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Royalty Pharma

                            Deal Size : $1,125.0 million

                            Deal Type : Agreement

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                            08

                            Alcyone Therapeutics

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed

                            Alcyone Therapeutics

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Biogen

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRxTM System for Ad...

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            June 27, 2023

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Biogen

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Alcyone Therapeutics

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed

                            Alcyone Therapeutics

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Biogen

                            Deal Size : $51.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Biogen and Alcyone Therapeutics Announce License and Collaboration Agreement to Evaluate a Novel Device to Imp...

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : $10.0 million

                            April 01, 2023

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Biogen

                            Deal Size : $51.0 million

                            Deal Type : Collaboration

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                            10

                            Biogen

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed

                            Biogen

                            U.S.A arrow
                            Peptide Process CMC
                            Not Confirmed
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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Data Presented at Cure SMA Reveal Residual Unmet Needs in Young SMA Patients Treated With Gene Therapy and...

                            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.

                            Product Name : Spinraza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            June 15, 2022

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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