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Lead Product(s): Undisclosed
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: Discovery Platform Product Type: Small molecule
Recipient: Skyhawk Therapeutics
Deal Size: $2,240.0 million Upfront Cash: $40.0 million
Deal Type: Collaboration December 22, 2020
Details:
The collaboration leverages SkySTAR™ platform to discover and develop novel small molecule therapeutics that modulate RNA splicing which have the potential to transform the lives of patients with serious diseases.
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Lead Product(s): Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Kalydeco
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 05, 2020
Details:
KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland.
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Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 04, 2020
Details:
Data in seven patients from two ongoing Phase 1/2 trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition.
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Lead Product(s): VX-814
Therapeutic Area: Genetic Disease Product Name: VX-814
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 14, 2020
Details:
Phase 2 study of VX-814 in patients with alpha-1 antitrypsin deficiency discontinued based upon safety and pharmacokinetic data. Phase 2 study of VX-864 continues to enroll and dose patients.
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Lead Product(s): Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Kalydeco
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 25, 2020
Details:
Safety data from a cohort of the Phase 3 ARRIVAL study support treatment with KALYDECO in children ages four to <6 months with eligible mutations.
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Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Recipient: CRISPR Therapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 22, 2020
Details:
European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).
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Lead Product(s): LNP based mRNA Therapeutics
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Discovery Product Type: Cell and Gene therapy
Recipient: Moderna Therapeutics
Deal Size: $455.0 million Upfront Cash: $75.0 million
Deal Type: Collaboration September 16, 2020
Details:
The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator protein to be produced.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Trikafta
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 10, 2020
Details:
The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Trikafta
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 01, 2020
Details:
U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor).
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Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor
Therapeutic Area: Genetic Disease Product Name: Kaftrio
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 21, 2020
Details:
E.C has granted marketing authorization for KAFTRIO® in a combination regimen with ivacaftor to treat people with cystic fibrosis ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.
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