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[{"orgOrder":0,"company":"Amarna Therapeutics","sponsor":"C4 holding BV","pharmaFlowCategory":"D","amount":"$5.7 million","upfrontCash":"Undisclosed","newsHeadline":"Amarna Therapeutics Secures Additional \u20ac5 ($5.7) Million to Advance Its Transformative Gene Therapy Platform Towards Clinical Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Amarna Therapeutics"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Receives FDA\u2019s IND Clearance and Fast Track Designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Azafaros"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
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Details:
AZ-3102 is a, novel small molecule, candidate developed for Lysosomal Storage Disorders with neurological involvement. AZ-3102 is an oral, brain penetrant azasugar, having dual mode of action by inhibiting two key enzymes which modulate the metabolism of glycosphingolipids.
Lead Product(s):
AZ-3102
Therapeutic Area: Genetic Disease
Product Name: AZ-3102
Highest Development Status: IND Enabling
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
January 05, 2023
Details:
The funds will enable Amarna to advance its lead gene therapy AMA005, for the treatment of hemophilia B towards a first-in-human clinical trial, and to progress its R&D pipeline in selected autoimmune diseases and chronic inflammation.
Lead Product(s):
AMA005
Therapeutic Area: Genetic Disease
Product Name: AMA005
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
C4 holding BV
Deal Size: $5.7 million
Upfront Cash: Undisclosed
Deal Type: Financing
December 01, 2021