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Find Drugs for Genetic Disease in Preclinical Development

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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).

            Vita Therapeutics

            • Development Update

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            Lead Product(s): VTA-110

            Therapeutic Area: Genetic Disease Product Name: VTA-110

            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 23, 2020

            Details:

            VTA-110, is a potential first-in-class allogenic iPSC-based therapy that has shown the ability to repair and regenerate healthy muscle in preclinical studies and has the potential to benefit patients with DMD.

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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).

            Erasmus University Medical Center

            • Development Update

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            Lead Product(s): Hematopoietic Stem Cell 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 July 08, 2020

            Details:

            Findings suggest that blood stem cell gene therapy, in which a patient’s own stem cells are modified to produce the acid alpha-glucosidase (GAA) enzyme, may be a promising approach for treating people with classic infantile Pompe disease.

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            Lead Product(s): FIX-Gene Therapy

            Therapeutic Area: Genetic Disease Product Name: CB 2679d-GT

            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 08, 2020

            Details:

            The new data include a direct comparison between CB 2679d-GT and the Padua variant in a mouse model of hemophilia B.

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            Lead Product(s): Imlifidase

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Sarepta Therapeutics

            Deal Size: $407.5 million Upfront Cash: $10.0 million

            Deal Type: Licensing Agreement July 02, 2020

            Details:

            Hansa Biopharma Granted Sarepta an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy treatment in Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD).

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            Lead Product(s): SPVN06

            Therapeutic Area: Genetic Disease Product Name: SPVN06

            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 June 30, 2020

            Details:

            SPVN06 uses a gene therapy-based approach independent of mutated genes and by a single subretinal injection of proprietary neurotrophic factors, aims at stopping and preventing the degeneration of photoreceptors leading to blindness.

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            Lead Product(s): mRNA-3745

            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 June 02, 2020

            Details:

            Preclinical data show that 3’-idT-stabilized mRNA encoding phenylalanine hydroxylase (PAH), the enzyme missing or dysfunctional in the metabolic disorder phenylketonuria (PKU), supports sustained reduction of serum phenylalanine levels in a PKU mouse model.

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            Lead Product(s): CRISPR/Cas9 based gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: REGENERON

            Deal Size: $100.0 million Upfront Cash: $70.0 million

            Deal Type: Agreement June 01, 2020

            Details:

            Under the amended agreement, Regeneron will have rights to discover and develop CRISPR/Cas9-based therapeutic products for an additional five in vivo liver targets, for a total of up to 15 targets.

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