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Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development

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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: 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 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 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): Gallium Citrate

            Therapeutic Area: Genetic Disease Product Name: AR-501

            Highest Development Status: Phase I/ Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 22, 2020

            Details:

            AR-501 is being developed as a once-per-week dosing regimen that is self-administered using a hand-held nebulizer device. Phase 1 portion of the Phase 1/2a trial, 48 healthy volunteers were treated with either single or multiple weekly dose regimens with no adverse effects.

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            Lead Product(s): BS01

            Therapeutic Area: Genetic Disease Product Name: BS01

            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 16, 2020

            Details:

            This is an important step toward bringing BS01 forward as a treatment to enable patients with retinal degenerative diseases to detect light, motion, and shapes, with the ultimate goal of restoring eyesight.

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            Lead Product(s): AT-007

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 15, 2020

            Details:

            Applied Therapeutics announced additional supportive biomarker efficacy and safety data for AT-007 in Galactosemia at 40 mg/kg. The company also announced initiation of the AT-007 pediatric trial, ACTION-Galactosemia Kids, in children age 2 to 17.

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            Lead Product(s): LentiGlobin

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 12, 2020

            Details:

            LentiGlobin™ gene therapy for adult and adolescent patients with sickle cell disease (SCD) show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS).

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            Lead Product(s): AXO-AAV-GM1

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 08, 2020

            Details:

            Axovant has completed enrollment in the low-dose cohort of the Phase 1/2 study for Type II (late infantile and juvenile onset) GM1 patients evaluating safety, tolerability, and exploratory measures of efficacy at a dose of 1.5x1013 vg/kg delivered intravenously.

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            Lead Product(s): SRP-9003

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 08, 2020

            Details:

            SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4). The therapy resulted in a mean signal intensity of 73% in the high-dose group compared to normal control. Mean beta-sarcoglycan expression of 62% in the high-dose cohort.

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            Lead Product(s): SRP-9003

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 05, 2020

            Details:

            Sarepta Therapeutics presents new data from the ongoing study of SRP-9003. Results will include expression and safety results from the high dose cohort and one-year functional results from the low dose cohort.

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            Lead Product(s): LentiGlobin

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Bluebird bio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement May 21, 2020

            Details:

            The two subsidiaries, Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma, will be in charge of late-clinical development and manufacturing of LentiGlobin at facilities in Germany (Apceth) and the U.S. (HCATS).

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