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[{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA offers CRISPR, Vertex drug program VIP status for its comeback play","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CRISPR Therapeutics"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Fast Track Designation for BCX9930 in PNH","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation for BCX9930 in PNH","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst\u2019s Oral Factor D Inhibitor, BCX9930, Shows Clinical Benefit as Monotherapy Through 400 mg bid in Treatment-na\u00efve PNH Patients","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Forms Scientific Advisory Board to Guide Global Development of CAN106 in Complement-mediated Diseases","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"}]
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CAN106 achieved rapid, dose-dependent reductions in both free C5 complement, a measure of serum hemolytic activity, within 24 hours of dosing. All subjects in two highest dose groups of CAN106 showed >99% reduction in free C5 and ≥90% inhibition of CH50.
Lead Product(s):
CAN106
Therapeutic Area: Hematology
Product Name: CAN106
Highest Development Status: Phase I/ Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
July 06, 2022
Details:
The FDA has granted both Fast Track status and Orphan Drug Designation to BCX9930 for PNH. BioCryst has confirmed meetings with regulators in the 4th quarter of 2020 to discuss the advanced development program for BCX9930.
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
Highest Development Status: Phase I/ Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 30, 2020
Details:
BioCryst Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
Highest Development Status: Phase I/ Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 31, 2020
Details:
U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
Highest Development Status: Phase I/ Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 03, 2020
Details:
CTX001 has received Orphan Drug Designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the EMA for sickle cell disease and transfusion-dependent beta thalassemia .
Lead Product(s):
CTX001
Therapeutic Area: Hematology
Product Name: Undisclosed
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
May 11, 2020