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

A collaboration with SCORR Marketing

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            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 26, 2020

            Details:

            All eligible patients who completed the Phase I trial entered the Extension Protocol, an important signal of how patients perceive benefits of receiving Trappsol® Cyclo™ intravenously.

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            Lead Product(s): MRT5005

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 26, 2020

            Details:

            First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

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            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 20, 2020

            Details:

            Data from the current study combined with those of the companion Phase I study will be used to inform the design of the Phase III global pivotal trial.

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            Lead Product(s): GTX-102

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 14, 2020

            Details:

            The Phase 1/2 open-label, multiple-dose, dose-escalating study will evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102.

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            Lead Product(s): CTX001

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2020

            Details:

            Enrollment ongoing in clinical trials of CTX001™ for patients with severe hemoglobinopathies

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            Lead Product(s): FLT190

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2020

            Details:

            As of the data cutoff date, there have been no infusion-related adverse events. Two Grade 1 or 2 serious adverse events have been reported in the study.

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            Lead Product(s): mRNA-3704

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II Product Type: Vaccine

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2020

            Details:

            mRNA-3704 is Moderna’s first rare disease program to enter clinical studies.

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            Lead Product(s): AVR-RD-04

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II 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:

            Early data trends from first patient dosed in the AVR-RD-04 investigational gene therapy program for cystinosis show improvements across multiple measures.

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            Lead Product(s): AVR-RD-04

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 04, 2020

            Details:

            The company is currently conducting studies on Lentiviral gene therapy to treat Lysosomal disorders and AVR-RD-01 as investigational lentiviral gene therapy.

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            Lead Product(s): RP-L201

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Lonza

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Partnership January 30, 2020

            Details:

            The agreement covers development and manufacturing of RP-L201, a phase 1/2 candidate for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).

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