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Lead Product(s): Tideglusib
Therapeutic Area: Genetic Disease Product Name: AMO-02
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 09, 2020
Details:
Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending marketing approval. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world.
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Lead Product(s): FLT180a
Therapeutic Area: Genetic Disease Product Name: FLT180a
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Undisclosed
Deal Size: $100.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering July 17, 2020
Details:
The company's lead candidate, FLT180a, an investigational gene therapy medicinal product candidate for the treatment of hemophilia B, is currently in a Phase 1/2 trial and has dosed 10 patients as of June 15, 2020.
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Lead Product(s): FLT180a
Therapeutic Area: Genetic Disease Product Name: FLT180a
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Novo Holdings
Deal Size: $120.0 million Upfront Cash: Undisclosed
Deal Type: Series C Financing June 30, 2020
Details:
Freeline expects to use the proceeds from the financing to bring its lead program in Haemophilia B into a pivotal trial, as well as to continue its Phase 1/2 clinical program for Fabry Disease and further progress its pipeline programs for Gaucher Disease and Haemophilia A.
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Lead Product(s): FLT180a
Therapeutic Area: Genetic Disease Product Name: FLT180a
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2020
Details:
Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients.
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Lead Product(s): OTL-101
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 07, 2020
Details:
Orchard Therapeutics to establish OTL-200, OTL-103, OTL-203 and OTL-201 programs as top near-term priorities and reduce investment in OTL-101and OTL-300.
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