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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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            Lead Product(s): ATR-12

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 May 27, 2020

            Details:

            If ATR-12 is approved, Azitra would be eligible to receive a priority review voucher, which can be used for priority review of a drug marketing application for another product being developed by the company.

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            Lead Product(s): ETX101

            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 May 19, 2020

            Details:

            ETX101, a gene therapy developed by Encoded Therapeutics to restore the levels of NaV1.1-alpha specifically in GABAergic neurons, is currently progressing toward clinical development for SCN1A-positive Dravet patients.

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            Lead Product(s): AAV-TT

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Cure Sanfilippo Foundation

            Deal Size: $0.4 million Upfront Cash: Undisclosed

            Deal Type: Funding May 15, 2020

            Details:

            The funding will help support creation of plasmid (a critical component of gene therapy drug production), as well as the required in-vivo efficacy study in the MPS IIIC mouse model.

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            Lead Product(s): TANGO antisense oligonucleotides

            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 May 12, 2020

            Details:

            The data demonstrate in-vitro and in-vivo proof-of-concept for TANGO ASOs in an ocular disease. First proof-of-concept data for TANGO antisense oligonucleotides (ASOs) in an ocular disease.

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            Lead Product(s): FORCE-M23D

            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 May 12, 2020

            Details:

            Enhanced dystrophin expression in multiple muscle tissues and sustained functional benefit in Duchenne Muscular Dystrophy disease model following single low dose.

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            Lead Product(s): PBKR03

            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 May 12, 2020

            Details:

            Preclinical data from University of Pennsylvania’s Gene Therapy Program shows that a single injection of AAVhu68 carrying a functional GALC gene improved all parameters of Krabbe disease.

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            Lead Product(s): ARCT-810

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 April 13, 2020

            Details:

            Two clinical trials for company's ARCT-810, being developed to treat ornithine transcarbamylase (OTC) deficiency have been approved by U.S FDA, and New Zealand Medicines, and Medsafe.

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            Lead Product(s): Stereopure oligonucleotides

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 February 18, 2020

            Details:

            Wave’s investigational allele-selective stereopure oligonucleotides is designed to preferentially target the mutant huntingtin (mHTT) mRNA transcript associated with a single nucleotide polymorphism.

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