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Find FDA Investigational New Drug (IND) Submissions for Genetic Disease

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            Lead Product(s): HST5040

            Therapeutic Area: Genetic Disease Product Name: HST5040

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 24, 2020

            Details:

            The U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to conduct a phase 2 clinical trial of HST5040, an oral small molecule drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA).

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            Lead Product(s): 4D-310

            Therapeutic Area: Genetic Disease Product Name: 4D-310

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Viking Global Investors

            Deal Size: $75.0 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing June 16, 2020

            Details:

            Funds to be used to advance 3 precision-guided AAV gene therapy candidates through initial clinical proof-of-concept, to advance pre-clinical product pipeline, proprietary next-generation Therapeutic Vector Evolution platform, and to expand GMP manufacturing capabilities.

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            Lead Product(s): MGTA-117

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Magenta Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration June 15, 2020

            Details:

            This non-exclusive research and clinical collaboration agreement aims to evaluate the potential utility of MGTA-117, Magenta’s novel targeted ADC for conditioning of patients with sickle cell disease and beta-thalassemia receiving Beam’s base editing therapies.

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            Lead Product(s): EDIT-301

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 12, 2020

            Details:

            EDIT-301 contains CD34+ hematopoietic stem cells from sickle patients that are edited at the HBG1/2 promoter in the beta-globin locus using Cas12a (also known as Cpf1) to induce fetal hemoglobin (HbF) where HbF-inducing mutations occur naturally.

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            Lead Product(s): PBGM01

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 21, 2020

            Details:

            USFDA has granted Rare Pediatric Disease designation to PBGM01 broadly for the treatment of GM1 gangliosidosis. PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis.

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            Lead Product(s): INZ-701

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 14, 2020

            Details:

            Inozyme is preparing to seek regulatory approval to initiate a clinical trial in patients with ENPP1 deficiency and a separate clinical trial in patients with ABCC6 deficiency.

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            Lead Product(s): LB-001

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 11, 2020

            Details:

            LogicBio's presentation will highlight results of treatment of Juvenile Mice with methylmalonic Acidemia (MMA) by targeted integration of MMUT into Albumin using a Promoterless AAV Vector.

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            Lead Product(s): DiNA-001

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: BioMarin Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement May 03, 2020

            Details:

            The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy. Additionally, the companies will collaborate on several of DiNAQOR's other pipeline programs.

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            Lead Product(s): TGTX-101

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: PBM Capital

            Deal Size: $30.0 million Upfront Cash: Undisclosed

            Deal Type: Financing April 29, 2020

            Details:

            Financing would assist in meeting Taysha's goal of advancing its 15 AAV gene therapy programs pipeline with options for another four programs targeting central nervous system diseases.

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            Lead Product(s): 4D-310

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 29, 2020

            Details:

            Oral presentations to include data on 4D-310 Product Candidate for Fabry Disease and 4D-A101 Aerosol Vector for Cystic Fibrosis and other Lung Diseases.

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