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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

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            Lead Product(s): Olipudase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

            Details:

            Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

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            Lead Product(s): Leriglitazone

            Therapeutic Area: Genetic Disease Product Name: MIN-102

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: European Investment Bank

            Deal Size: $29.0 million Upfront Cash: Undisclosed

            Deal Type: Funding October 30, 2020

            Details:

            The EU bank will grant long-term financing to Minoryx to drive the company’s research and development activities in orphan genetic diseases which includes Pivotal Phase 2/3 ADVANCE study, Phase 2 NEXUS study and Phase 2 FRAMES study.

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            Lead Product(s): LYS-SAF302

            Therapeutic Area: Genetic Disease Product Name: AAVrh10-h.SGSH

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 26, 2020

            Details:

            AAVance clinical trial is a global Phase 2/3 gene therapy trial for the treatment of Mucopolysaccharidosis. The company remains committed to the LYS-SAF302 development program and the Sanfilippo patient community.

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            Lead Product(s): Elamipretide

            Therapeutic Area: Genetic Disease Product Name: MTP-131

            Highest Development Status: Phase II/ Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 19, 2020

            Details:

            The poster titled "TAZPOWER Analysis: Elamipretide Significantly Improves Disease Symptomatology versus Natural History Controls in Barth Syndrome," shows the importance of longitudinal natural history data in understanding and validating clinical trial outcomes for Barth.

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            Lead Product(s): LYS-SAF302

            Therapeutic Area: Genetic Disease Product Name: AAVrh10-h.SGSH

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 15, 2020

            Details:

            Company has announced death of a patient in the AAVance study , a phase 2/3 international clinical study evaluating gene therapy treatment of Mucopolysaccharidosis type IIIA (MPS IIIA, known as Sanfilippo Syndrome type A) .

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Zygel

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 15, 2020

            Details:

            New data demonstrate that in patients diagnosed with FXS with a fully methylated FMR1 gene significantly more patients who received Zygel achieved a clinically meaningful improvement in their behavioral symptoms compared to patients who received placebo.

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            Lead Product(s): ARO-AAT

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $1,040.0 million Upfront Cash: $300.0 million

            Deal Type: Licensing Agreement October 08, 2020

            Details:

            Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the U.S under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global marketing strategy and receive an exclusive license to commercialize ARO-AAT.

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            Lead Product(s): Leriglitazone

            Therapeutic Area: Genetic Disease Product Name: MIN-102

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sperogenix Therapeutics

            Deal Size: $78.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement September 23, 2020

            Details:

            Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition.

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            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $650.0 million Upfront Cash: $650.0 million

            Deal Type: Agreement July 20, 2020

            Details:

            Risdiplam, to be marketed by Roche, is an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy (SMA).

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            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

            Details:

            Orphazyme has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).

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