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II"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline Reports Positive Initial Clinical Data from First Cohort of Phase 1\/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Syncona","pharmaFlowCategory":"D","amount":"$28.3 million","upfrontCash":"$28.3 million","newsHeadline":"Syncona to Acquire Freeline Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New 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            Details:

            Through the acquisition, Syncona gains access to FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, currently in Phase 1/2 clinical trial in people with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Syncona

            Deal Size: $28.3 million Upfront Cash: $28.3 million

            Deal Type: Acquisition February 20, 2024

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            Through the acquisition, Syncona gains access to FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, currently in Phase 1/2 clinical trial in people with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Syncona

            Deal Size: $28.3 million Upfront Cash: $28.3 million

            Deal Type: Acquisition November 22, 2023

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            FLT201 is an adeno-associated virus (AAV) gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

            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 October 04, 2023

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            FLT201 is an adeno-associated virus (AAV) gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

            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 June 26, 2023

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            FLT201 is an adeno-associated virus (AAV) gene therapy enhacer of GCase level that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

            Lead Product(s): FLT201

            Therapeutic Area: Genetic Disease Product Name: FLT201

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 22, 2023

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            Under the terms of the agreement, Freeline gains rights to develop and commercialise AAV-based gene therapies for certain cardiac conditions using key targets identified by Forcefield.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Recipient: Forcefield Therapeutics

            Deal Size: $0.6 million Upfront Cash: Undisclosed

            Deal Type: Agreement April 04, 2023

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            FLT190 is designed to generate durable increases in α-Gal A levels and reduce substrate accumulation, with the aim of providing a one-time treatment that can stop disease progression and improve outcomes. It s currently being investigated for fabry disease.

            Lead Product(s): FLT190

            Therapeutic Area: Genetic Disease Product Name: FLT190

            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 January 19, 2023

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            FLT190 is a gene therapy candidate designed to generate durable plasma α-Gal A levels above the normal range to enable sufficient enzyme activity in relevant tissues with a one-time treatment.

            Lead Product(s): FLT190

            Therapeutic Area: Genetic Disease Product Name: FLT190

            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 October 04, 2022

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            FLT180a (verbrinacogene setparvovec), uses a potent AAVS3 capsid rationally designed for effective targeting and transduction of liver cells and containing an expression cassette encoding a gain of function Padua variant of human factor IX (FIX).

            Lead Product(s): Verbrinacogene Setparvovec

            Therapeutic Area: Genetic Disease Product Name: FLT180a

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: University College London

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 21, 2022

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            B-LIEVE is a Phase 1/2 dose confirmation trial of FLT180a (verbrinacogene setparvovec) using a short course of prophylactic immune management with the goal of normalizing FIX levels in patients with severe and moderately severe hemophilia B.

            Lead Product(s): Verbrinacogene Setparvovec

            Therapeutic Area: Genetic Disease Product Name: FLT180a

            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 June 24, 2022

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