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Biogen

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Biogen Announces First Patient Treated in Respond Study Evaluating Benefit of Spinraza® in Patients Treated with Zolgensma®

Details:

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 IV Product 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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Biogen

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Biogen to Initiate Phase 4 Respond Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma

Details:

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 IV Product 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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Vertex Pharmaceuticals

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Vertex Pharmaceuticals Reports Positive Phase 3 Study Results for TRIKAFTA® in People Ages 12 and Older With Cystic Fibrosis

Details:

The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508del mutation.

Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

Therapeutic Area: Genetic Disease Product Name: Trikafta

Highest Development Status: Phase IV Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 20, 2020

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