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            Lead Product(s): REV-0100

            Therapeutic Area: Genetic Disease Product Name: REV-0100

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 28, 2020

            Details:

            REV-0100 is a potential therapy for patients with Stargardt disease, designed to bind and clear a toxic lipid called lipofuscin. REV-0100 was developed from research from Weill Cornell Medicine in New York City, and is covered by a granted patent.

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            Lead Product(s): MMA-101,ImmTOR

            Therapeutic Area: Genetic Disease Product Name: MMA-101

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

            Partner/Sponsor/Collaborator: Asklepios BioPharmaceutical

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 20, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations.

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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): 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): 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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            Lead Product(s): KB407

            Therapeutic Area: Genetic Disease Product Name: KB407

            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 August 17, 2020

            Details:

            U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to KB407, currently in preclinical development for the treatment of cystic fibrosis.

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