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            Lead Product(s): HGB-206

            Therapeutic Area: Genetic Disease Product Name: HGB-206

            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 February 16, 2021

            Details:

            Following a report that a patient treated more than five years earlier in Group A of HGB-206 had been diagnosed with AML, bluebird bio temporarily halted the studies for HGB-206 and HGB-210 in accordance with the clinical trial protocols.

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            Lead Product(s): FLT180a

            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 February 08, 2021

            Details:

            Under the modified clinical development plan, Freeline now plans to conduct dose confirmation in a FLT180a Phase 1/2 trial instead of in the Phase 2b part of the previously planned Phase 2b/3 pivotal trial.

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            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 January 26, 2021

            Details:

            Abstract entitled "Follow-up on a Novel Adeno Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)" will be presented at the conference.

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            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 December 14, 2020

            Details:

            Study results show durable Factor IX activity up to a period of nearly three years, with no bleeds reported requiring FIX supplementation, and support selection of a dose and immune management regimen that has the potential to deliver FIX activity in the normal range.

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            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 08, 2020

            Details:

            Preliminary results from first patient treated with OTL-201 show over expression of the therapeutic enzyme and decrease in substrate levels three months following gene therapy.

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            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 05, 2020

            Details:

            The oral presentation will highlight data from the first patient treated in the ongoing proof-of-concept study of OTL-201, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA.

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            Lead Product(s): FLT180a

            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 July 13, 2020

            Details:

            Updated FLT180a data in the B-AMAZE study in severe Haemophilia B patients demonstrated the potential for sustained normal FIX activity levels.