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Find Drugs for Genetic Disease in Phase II Clinical Development in UNITED STATES

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            Lead Product(s): Lenabasum

            Therapeutic Area: Genetic Disease Product Name: JBT-101

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 06, 2020

            Details:

            The CF-002 Phase 2b trial did not meet the primary endpoint of a statistically significant reduction in rate of new PEx per subject per 28 weeks. Lenabasum treatment had a favorable safety profile and was well-tolerated.

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            Lead Product(s): Apitegromab

            Therapeutic Area: Genetic Disease Product Name: SRK-015

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2020

            Details:

            The SRK-015 clinical development poster will showcase data from the Phase 1 healthy volunteer trial and baseline characteristics, demographics from the TOPAZ Phase 2 proof-of-concept trial evaluating SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy.

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            Lead Product(s): Soticlestat

            Therapeutic Area: Genetic Disease Product Name: TAK-935

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2020

            Details:

            Results from signal-finding, pilot Phase 2 open-label ARCADE study and ENDYMION long-term extension study in CDKL5 deficiency disorder (CDD) and Dup15q syndrome (Dup15q) show seizure frequency reduction over time .

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            Lead Product(s): Allogeneic cardiosphere-derived cells

            Therapeutic Area: Genetic Disease Product Name: CAP-1002

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 23, 2020

            Details:

            Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and the cytokine storm associated with COVID-19.

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            Lead Product(s): Pepinemab

            Therapeutic Area: Genetic Disease Product Name: VX15/2503

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 22, 2020

            Details:

            Key cognitive endpoints trending towards but did not reach statistical significance in early manifest population. Overall study does not meet pre-specified co-primary endpoints.

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Zygel

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 17, 2020

            Details:

            U.S. Food and Drug Administration has granted orphan drug designation for cannabidiol (CBD) for use in treating 22q11.2 deletion syndrome (22q).

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            Lead Product(s): ARO-AAT

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 16, 2020

            Details:

            The results show clear evidence of a meaningful pharmacodynamic effect by ARO-AAT, leading to improvements in relevant biomarkers, including substantial reductions in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer.

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            Lead Product(s): MS1819

            Therapeutic Area: Genetic Disease Product Name: MS1819

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 15, 2020

            Details:

            Results from the Phase 2 OPTION clinical trial demonstrates that the non-porcine MS1819 lipase is well-tolerated with no clinically significant safety signals at the 2g daily dose.