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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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            Lead Product(s): AAV-based engineered transgene

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 11, 2020

            Details:

            The U.S FDA has granted Neurogene the Orphan Drug Designation for its adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease.

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 11, 2020

            Details:

            U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease.

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            Lead Product(s): Antibody-oligonucleotide conjugates

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Vida Ventures

            Deal Size: $115.0 million Upfront Cash: Undisclosed

            Deal Type: Financing August 10, 2020

            Details:

            The financing accelerates the development of Dyne’s pipeline of muscle disease therapies, including its lead programs in myotonic dystrophy type 1, Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy, as well as additional indications.

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            Lead Product(s): AAV-hNR2E3

            Therapeutic Area: Genetic Disease Product Name: OCU400

            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 10, 2020

            Details:

            U.S. Food and Drug Administration (FDA) granted Ocugen the fourth Orphan Drug Designation (ODD) for OCU400 in the treatment of PDE6B gene mutation-associated retinal diseases. In pre-clinical studies, OCU400 has demonstrated improved vision signals in the retina.

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            Lead Product(s): TSHA-101

            Therapeutic Area: Genetic Disease Product Name: TSHA-101

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

            Partner/Sponsor/Collaborator: Fidelity Management & Research Company

            Deal Size: $95.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing August 05, 2020

            Details:

            Financing will go in advancing initial cohort of lead programs into the clinic, accelerate progress on anticipated IND submissions, build a commercially scalable GMP manufacturing facility and continue development of the company’s extensive portfolio.

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            Lead Product(s): AAV-hNR2E3

            Therapeutic Area: Genetic Disease Product Name: OCU400

            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 July 27, 2020

            Details:

            Third orphan drug designation for the same product, OCU400, is unique in Ophthalmology gene therapy and demonstrates its potential to treat many Inherited Retinal Degenerative diseases (IRDs).

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            Lead Product(s): TTX-3360

            Therapeutic Area: Genetic Disease Product Name: TTX-3360

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 21, 2020

            Details:

            Recent experiments by Triplet demonstrated that a single dose of TTX-3360 induced significant knockdown of the target gene mRNA at 29 days in the cortex (>75%) and caudate (>45%) in non-human primates.

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            Lead Product(s): Ascorbic Acid,Glutathione,Bicarbonate

            Therapeutic Area: Genetic Disease Product Name: ARINA-1

            Highest Development Status: Preclinical 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 paper provides an overview of the impact of ARINA-1 on mucus transport and shows that the ARINA-1 moves mucus significantly better than currently available options in cystic fibrosis (CF).

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