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

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            Lead Product(s): SIG-001

            Therapeutic Area: Genetic Disease

            Highest Development Status: IND Enabling Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Canada Pension Plan Investment Board

            Deal Size: $80.3 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing March 17, 2020

            Details:

            The funding will support the first-in-human clinical trial of Sigilon’s novel encapsulated cell therapy for hemophilia A.

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            Lead Product(s): LB-001

            Therapeutic Area: Genetic Disease

            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 February 10, 2020

            Details:

            The Company submitted the IND in January 2020 to support the initiation of a Phase 1/2 clinical trial in patients with MMA.

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            Lead Product(s): PBGM01

            Therapeutic Area: Genetic Disease

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $125.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 03, 2020

            Details:

            Passage Bio intend to use the net proceeds to advance its lead products in clinical trials, to advance its discovery and candidate selection stage programs, and for general corporate purposes.

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            Lead Product(s): GTX-102

            Therapeutic Area: Genetic Disease

            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 January 15, 2020

            Details:

            GeneTx’s Phase 1/2 study, named KIK-AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020.

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            Lead Product(s): BMN 307

            Therapeutic Area: Genetic Disease

            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 January 13, 2020

            Details:

            BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program and actively preparing regulatory submissions.

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            Lead Product(s): L-Homocysteine

            Therapeutic Area: Genetic Disease

            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 January 13, 2020

            Details:

            Aeglea BioTherapeutics expects to initiate a Phase 1/2 trial in the second quarter of 2020.

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            Lead Product(s): LB-001

            Therapeutic Area: Genetic Disease

            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 January 10, 2020

            Details:

            LogicBio Therapeutics has submitted an IND application with the U.S. FDA to initiate a Phase 1/2 trial of LB-001, for the treatment of methylmalonic acidemia (MMA).

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            Lead Product(s): PR001

            Therapeutic Area: Genetic Disease

            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 January 09, 2020

            Details:

            Company Advancing its Phase 1/2 Clinical Trial for the Treatment of Neuronopathic Gaucher Disease and Expects to Initiate Patient Dosing in First Half of 2020.