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            Lead Product(s): Edasalonexent

            Therapeutic Area: Musculoskeletal

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Oppenheimer & Co.

            Deal Size: $23.0 million Upfront Cash: Undisclosed

            Deal Type: IPO January 30, 2020

            Details:

            Catabasis intends to use the net proceeds from the offering for clinical trial and other research and development activities; initial commercialization preparations; and for working capital.

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            Lead Product(s): Losmapimod

            Therapeutic Area: Musculoskeletal

            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 January 29, 2020

            Details:

            The Phase 1 clinical trial of losmapimod in patients with FSHD, indicated that losmapimod was generally well tolerated and achieved dose-dependent concentrations in plasma and muscle.

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            Lead Product(s): Palovarotene

            Therapeutic Area: Musculoskeletal

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 24, 2020

            Details:

            Ipsen to conduct further assessment of the complete data set and work with the regulatory authorities, based on encouraging therapeutic activity signals observed in preliminary post-hoc analyses.

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            Lead Product(s): Cad-1883

            Therapeutic Area: Musculoskeletal

            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 January 23, 2020

            Details:

            The Phase 2 trial is a multicenter, randomized, placebo-controlled study that will evaluate the safety and efficacy of CAD-1883. Efficacy outcome measures.

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            Lead Product(s): MIV-711

            Therapeutic Area: Musculoskeletal

            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 January 20, 2020

            Details:

            The study demonstrated significant effect on changes in bone and cartilage degradation after six months of treatment with MIV-711.

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            Lead Product(s): Burosumab

            Therapeutic Area: Musculoskeletal

            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 January 14, 2020

            Details:

            The sBLA package includes data from two single-arm Phase 2 studies including 14 adult patients conducted by Ultragenyx in the U.S. and 13 adult patients conducted by Kyowa Kirin in Japan.

            Harvard Stem Cell Institute

            • Deals

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            Lead Product(s): CRISPR/Cas9 genome

            Therapeutic Area: Musculoskeletal

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: Sarepta Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration January 13, 2020

            Details:

            As per collaboration agreement, the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein.

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            Lead Product(s): GNT0004

            Therapeutic Area: Musculoskeletal

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Sarepta Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration January 09, 2020

            Details:

            CDMO’s capacity to produce AAV materials at large-scale will support Genethon and Sarepta’s project to take micro-dystrophin to clinical trials this year.

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            Lead Product(s): Hyaluronic Acid,Lidocaine

            Therapeutic Area: Musculoskeletal

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Revance Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement January 09, 2020

            Details:

            Under the agreement Revance will gain immediate and exclusive rights to commercialize TEOXANE’s RHA® line of fillers in the U.S.

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            Lead Product(s): Edasalonexent

            Therapeutic Area: Musculoskeletal

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Duchenne UK

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership January 08, 2020

            Details:

            The partnership is to evaluate Edasalonexent a novel NF-kB inhibitor, in a Phase 2 Non-Ambulatory Duchenne Muscular Dystrophy Trial.

            PharmaCompass