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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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            Details:

            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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            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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            OTL-201 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IIIA. It uses a lentiviral vector to insert a functional copy of the human SGSH gene into a patient’s hematopoietic stem cells.

            Lead Product(s): OTL-201

            Therapeutic Area: Genetic Disease Product Name: OTL-201

            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 December 12, 2022

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            OTL-201 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patient’s hematopoietic stem cells.

            Lead Product(s): OTL-201

            Therapeutic Area: Genetic Disease Product Name: OTL-201

            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 03, 2022

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            Details:

            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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            Details:

            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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            Details:

            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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            Details:

            FLT180a (verbrinacogene setparvovec) candidate uses a potent, rationally designed AAVS3 capsid 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: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 14, 2022

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