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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: Phase IVProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 06, 2024

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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Biogen

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 27, 2023

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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Royalty Pharma

Deal Size: $1,125.0 million Upfront Cash: $500.0 million

Deal Type: Agreement January 09, 2023

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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Biogen

Deal Size: $51.0 million Upfront Cash: $10.0 million

Deal Type: Collaboration January 04, 2023

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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 15, 2022

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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 14, 2022

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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.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 15, 2021

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Initial findings from the DEVOTE study suggest no new safety concerns and support continued development of a higher dose of SPINRAZA® (nusinersen).


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 19, 2021

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The RESPOND study aims to evaluate if Spinraza can benefit SMA patients who have experienced a ‘suboptimal’ response to Zolgensma (onasemnogene abeparvovec). It will be conducted at around 20 sites globally, with researchers aiming to enrol up to 60 children with SMA.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: Phase IVProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 08, 2021

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The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its continuous production of SMN protein may also benefit patients previously treated with gene therapy.


Lead Product(s): Nusinersen

Therapeutic Area: Genetic Disease Product Name: Spinraza

Highest Development Status: Phase IVProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 21, 2020

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