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Find Drugs for Genetic Disease in Preclinical Development

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            Lead Product(s): TSHA-102

            Therapeutic Area: Genetic Disease Product Name: TSHA-102

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 14, 2020

            Details:

            U.S. Food and Drug Administration for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug application for TSHA-102 to the FDA in 2021.

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            Lead Product(s): ET-01

            Therapeutic Area: Genetic Disease Product Name: ET-01

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

            Partner/Sponsor/Collaborator: 3H Health Investment

            Deal Size: $67.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing October 13, 2020

            Details:

            EdiGene has established four gene editing based platforms and is advancing its early stage programs into clinical development for patients with genetic diseases and cancer. Proceeds from the financing will be used to advance the company’s pipeline into clinics.

            The University of Manchester

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            Lead Product(s): Lentiviral gene therapy

            Therapeutic Area: Genetic Disease Product Name: AVR-RD-05

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

            Partner/Sponsor/Collaborator: Avrobio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement October 05, 2020

            Details:

            Gene therapy licensed from The University of Manchester, will be called as AVR-RD-05. Avrobio will make the therapy by modifying patient’s own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of functional enzymes.

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            Lead Product(s): SPIRO-2101

            Therapeutic Area: Genetic Disease Product Name: SPIRO-2101

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 24, 2020

            Details:

            SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator.

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            Lead Product(s): CRN04777

            Therapeutic Area: Genetic Disease Product Name: CRN04777

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 21, 2020

            Details:

            U.S. Food and Drug Administration has granted rare pediatric disease designation (“RPD”) for CRN04777, an investigational, orally available, nonpeptide somatostatin receptor type 5 (SST5) agonist being developed as a treatment for congenital hyperinsulinism.

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            Lead Product(s): AT-02

            Therapeutic Area: Genetic Disease Product Name: AT-02

            Highest Development Status: Preclinical Product Type: Peptide

            Partner/Sponsor/Collaborator: venBio Partners

            Deal Size: $25.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing September 14, 2020

            Details:

            The financing will allow Attralus to further validate their pan-amyloid imaging agent, AT-01, and advance two therapeutic candidates, AT-02 and AT-03, into clinical development for ATTR (transthyretin), AL (light chain) and potentially ALECT2 systemic amyloidosis.

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: The Column Group

            Deal Size: $50.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing September 10, 2020

            Details:

            Proceeds will be used to advance Casma’s First-in-Class TRPML1 agonist in muscular dystrophy and enable preclinical proof-of-concept for the Company’s novel autophagy degrader platform.

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            Lead Product(s): INV-101

            Therapeutic Area: Genetic Disease Product Name: INV-101

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Forbion

            Deal Size: $35.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing September 08, 2020

            Details:

            Proceeds will be used to develop Inversago’s best-in-class peripherally-acting CB1 inverse agonist candidate INV-101 to clinical proof-of-concept, and advance the research on other selected compounds.

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            Lead Product(s): AOC 1001

            Therapeutic Area: Genetic Disease Product Name: AOC 1001

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Myotonic Dystrophy Clinical Research Network

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration September 08, 2020

            Details:

            The collaboration focuses at supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1. END-DM1 is a non-interventional study designed and run by the Myotonic Dystrophy Clinical Research Network.

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            Lead Product(s): LB-100

            Therapeutic Area: Genetic Disease Product Name: LB-100

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Lixte Biotechnology

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration August 18, 2020

            Details:

            Collaboration will support the preclinical studies of the potential benefit of Lixte’s proprietary lead clinical compound, LB-100, in a mouse model of Angelman Syndrome (AS).

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