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[{"orgOrder":0,"company":"Dynacure","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dynacure Announces First Patient Dosed in Phase 1 \/ 2 'UNITE-CNM' Study of DYN101","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Dynacure"},{"orgOrder":0,"company":"Dynacure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dynacure Gets Rare Pediatric Disease Designation from U.S. FDA for DYN101 for Myotubular and Centronuclear Myopathies","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Dynacure"},{"orgOrder":0,"company":"Histogen","sponsor":"U.S. Department of Defense","pharmaFlowCategory":"D","amount":"$2.0 million","upfrontCash":"Undisclosed","newsHeadline":"Histogen Announces $2M Grant Award from the Department of Defense for Clinical Advancement of HST-003 for Cartilage Regeneration in the Knee","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Histogen"},{"orgOrder":0,"company":"SpineThera","sponsor":"Department of Defense","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"SpineThera Awarded $5 Million Grant from Department of Defense","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"SpineThera"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Avidity Biosciences Granted FDA Fast Track Designation for AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Avidity Biosciences"},{"orgOrder":0,"company":"Immunis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Immunis Recruits Individuals with Muscle Atrophy for Phase 1\/2a Clinical Trial","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Immunis"}]

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Immunis

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Immunis Recruits Individuals with Muscle Atrophy for Phase 1/2a Clinical Trial

Details:

IMM01-STEM, a secretome product derived from partially differentiated pluripotent stem cells that contains regenerative molecules, in participants with muscle atrophy related to knee osteoarthritis (KOA).

Lead Product(s): IMM01-STEM

Therapeutic Area: Musculoskeletal Product Name: IMM01-STEM

Highest Development Status: Phase I/ 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 January 24, 2023

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Avidity Biosciences

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Avidity Biosciences Granted FDA Fast Track Designation for AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy

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AOC 1020 aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in patients with FSHD. AOC 1020 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA.

Lead Product(s): AOC 1020

Therapeutic Area: Musculoskeletal Product Name: AOC 1020

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 January 18, 2023

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SpineThera

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SpineThera Awarded $5 Million Grant from Department of Defense

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The scope of work supported by this grant award includes nonclinical toxicity testing of SX600 and manufacturing process development and scale-up activities. Together, these activities will position SX600 for future Phase 3 clinical trials.

Lead Product(s): Dexamethasone

Therapeutic Area: Musculoskeletal Product Name: SX600

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

Partner/Sponsor/Collaborator: Department of Defense

Deal Size: $5.0 million Upfront Cash: Undisclosed

Deal Type: Funding April 26, 2022

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Histogen

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Histogen Announces $2M Grant Award from the Department of Defense for Clinical Advancement of HST-003 for Cartilage Regeneration in the Knee

Details:

The funding supports Phase 1/2 clinical trial of HST-003 for regeneration of cartilage in the knee led by Histogen. The trial is designed to evaluate HST-003 in combination with a microfracture procedure to treat recent focal cartilage defects in the knee caused by injury.

Lead Product(s): HST-003

Therapeutic Area: Musculoskeletal Product Name: HST-003

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

Partner/Sponsor/Collaborator: U.S. Department of Defense

Deal Size: $2.0 million Upfront Cash: Undisclosed

Deal Type: Funding September 16, 2020

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Dynacure

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Dynacure Gets Rare Pediatric Disease Designation from U.S. FDA for DYN101 for Myotubular and Centronuclear Myopathies

Details:

U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2), for the treatment of Myotubular and Centronuclear Myopathies (CNM).

Lead Product(s): Dynamin 101

Therapeutic Area: Musculoskeletal Product Name: DYN101

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 July 30, 2020

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Dynacure

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Dynacure Announces First Patient Dosed in Phase 1 / 2 'UNITE-CNM' Study of DYN101

Details:

This clinical trial, based on Ionis' technology platform, is the first investigational study testing an antisense medicine to treat Centronuclear Myopathies.

Lead Product(s): DYN101

Therapeutic Area: Musculoskeletal Product Name: Undisclosed

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 March 05, 2020

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