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

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            Lead Product(s): ARO-ENaC

            Therapeutic Area: Genetic Disease Product Name: ARO-ENaC

            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 28, 2020

            Details:

            Arrowhead Pharmaceutical's key opinion leader webinar will highlight clinical developments of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis.

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            Lead Product(s): CTX001

            Therapeutic Area: Genetic Disease Product Name: CTX001

            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 27, 2020

            Details:

            CRISPR Therapeutics continues to make substantial progress driving its multiple, ongoing clinical development programs. Enrollment in its immuno-oncology trials is ongoing, the compay has re-initiated dosing in its CTX001 trials.

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            Lead Product(s): rAAV2tYF-GRK1-RPGR

            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 July 22, 2020

            Details:

            AGTC is conducting clinical trials of adeno-associated virus-based gene therapies for rare diseases, including X-linked retinitis pigmentosa (XLRP).

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            Lead Product(s): AAV2/5-RPGR

            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 July 17, 2020

            Details:

            The interim data showed that low and intermediate doses of the investigational adeno-associated virus retinitis pigmentosa GTPase regulator (AAV-RPGR) were generally well-tolerated and indicated significant improvement in vision.

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            Lead Product(s): ABO-101

            Therapeutic Area: Genetic Disease Product Name: ABO-101

            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 16, 2020

            Details:

            ABO-101 is Abeona’s first-in-human gene therapy for Sanfilippo syndrome, and was designed to deliver a functional copy of the NAGLU gene using a harmless adeno-associated viral (AAV) transporter.

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            Lead Product(s): SPK-8011

            Therapeutic Area: Genetic Disease Product Name: SPK-8011

            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 12, 2020

            Details:

            All five patients in the 5x1011 vg/kg and 1×1012 vg/kg dose cohorts and seven participants in the 2×1012 vg/kg dose show an acceptable safety profile, stable & durable factor VIII expression and substantial improvement in annualized bleed rate after treatment with SPK-8011.

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            Lead Product(s): PTR-01

            Therapeutic Area: Genetic Disease Product Name: BBP-589

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

            Partner/Sponsor/Collaborator: Phoenix Tissue Repair

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 10, 2020

            Details:

            The poster presentation, which includes safety and tolerability data observed so far in patients enrolled in cohorts 1-3, will be delivered by Anna L. Bruckner, MD, associate professor of dermatology and pediatrics at University of Colorado School of Medicine.

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            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Genetic Disease Product Name: Trappsol Cyclo

            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 July 09, 2020

            Details:

            The presentations describe top line results from the Phase I trial, which is now closed, and interim results from the Phase I/II trial which is expected to be completed in early 2021.

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            Lead Product(s): AVR-RD-02

            Therapeutic Area: Genetic Disease Product Name: AVR-RD-02

            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 06, 2020

            Details:

            First patient has been dosed in the company’s GuardOne clinical trial, a Phase 1/2 investigational study evaluating AVR-RD-02 for Gaucher disease type 1. The company also dosed second patient in the ongoing Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.

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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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