[{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cad-1883","moa":"Calcium-sensitive potassium ion channel PAM","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Oral","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Celavet","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"OK100 stem cell","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Celavet","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"Celavet \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Celavet \/ Not Applicable"},{"orgOrder":0,"company":"Virpax Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Diclofenac Epolamine","moa":"COX","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Virpax Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Topical Spray","sponsorNew":"Virpax Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Virpax Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"RTW Investments","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Series C Financing","leadProduct":"Antibody-Oligonucleotide Conjugates","moa":"DMPK","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Avidity Biosciences","amount2":0.10000000000000001,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Avidity Biosciences \/ RTW Investments","highestDevelopmentStatusID":"5","companyTruncated":"Avidity Biosciences \/ RTW Investments"},{"orgOrder":0,"company":"Virpax Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Public Offering","leadProduct":"Diclofenac","moa":"COX","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Virpax Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0.040000000000000001,"dosageForm":"Topical Spray Film","sponsorNew":"Virpax Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"5","companyTruncated":"Virpax Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"MiMedx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Micronized Dehydrated Human Amnion Chorion Membrane","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"MiMedx","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"MiMedx \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"MiMedx \/ Not Applicable"},{"orgOrder":0,"company":"Virpax Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Diclofenac Epolamine","moa":"COX1\/COX2","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Virpax Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Topical Spray","sponsorNew":"Virpax Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Virpax Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Virpax Pharmaceuticals","sponsor":"Kindeva Drug Delivery","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Diclofenac Epolamine","moa":"COX1\/COX2","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Virpax Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"Virpax Pharmaceuticals \/ Kindeva","highestDevelopmentStatusID":"5","companyTruncated":"Virpax Pharmaceuticals \/ Kindeva"},{"orgOrder":0,"company":"SiSaf","sponsor":"CSSi LifeSciences","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"UNITED KINGDOM","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"SIS-101-ADO","moa":"CLCN7 gene mutation","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"SiSaf","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"SiSaf \/ CSSi LifeSciences","highestDevelopmentStatusID":"5","companyTruncated":"SiSaf \/ CSSi LifeSciences"},{"orgOrder":0,"company":"Epic Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Epic Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"Epic Bio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Epic Bio \/ Not Applicable"},{"orgOrder":0,"company":"Epic Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Epic Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"Epic Bio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Epic Bio \/ Not Applicable"},{"orgOrder":0,"company":"Arugula Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"SIG002","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Arugula Sciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Arugula Sciences \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Arugula Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Epic Bio","sponsor":"Solve FSHD","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"EPI-321","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Epic Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"Epic Bio \/ Solve FSHD","highestDevelopmentStatusID":"5","companyTruncated":"Epic Bio \/ Solve FSHD"},{"orgOrder":0,"company":"SiSaf","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"UNITED KINGDOM","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"SIS-101-ADO","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"SiSaf","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"SiSaf \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"SiSaf \/ Not Applicable"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ARO-DUX4","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"Arrowhead Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Arrowhead Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Celularity","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"PDA-002","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"Celularity","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Injection","sponsorNew":"Celularity \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Celularity \/ Not Applicable"},{"orgOrder":0,"company":"MMJ International Holding","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"MMJ-002","moa":"","graph1":"Musculoskeletal","graph2":"IND Enabling","graph3":"MMJ International Holding","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"","sponsorNew":"MMJ International Holding \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"MMJ International Holding \/ Not Applicable"}]

Find FDA Investigational New Drug (IND) Submissions for Musculoskeletal

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : Allocetra is being developed as a universal, off-the-shelf cell therapy designed to reprogram macrophages into their homeostatic state in patients with TMJ osteoarthritis.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 11, 2024

                          Lead Product(s) : Allocetra-OTS

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : MMJ-002 is a novel cannabis-derived medication, being investigated for the treatment of chorea associated with Huntington's disease (HD).

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 10, 2024

                          Lead Product(s) : MMJ-002

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : FDA has agreed to an amendment to the company’s investigational new drug (IND) application to evaluate ADI-001 in idiopathic inflammatory myopathy (IIM) and stiff person syndrome.

                          Product Name : ADI-001

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          October 16, 2024

                          Lead Product(s) : ADI-001

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PDA-002 is a placenta-derived mesenchymal-like adherent stromal cell therapy, which is being evaluated for the treatment of facioscapulohumeral muscular dystrophy.

                          Product Name : PDA-002

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 20, 2024

                          Lead Product(s) : PDA-002

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : ARO-DUX4 is an investigational RNAi therapeutic designed to reduce expression of the gene that encodes the human double homeobox 4 (DUX4) protein as a potential treatment for FSHD.

                          Product Name : ARO-DUX4

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          July 17, 2023

                          Lead Product(s) : ARO-DUX4

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : SIS-101-ADO is an siRNA that suppresses the expression of CLCN7, a mutant gene expressed by osteoclasts and other cell types responsible for causing ADO2, the RNA therapy restores bone mass and quality to nearly normal levels.

                          Product Name : SIS-101-ADO

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 15, 2023

                          Lead Product(s) : SIS-101-ADO

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : The investment will support development of Epic’s lead program EPI-321, a novel therapeutic that is designed to correct the epigenetic alterations in the D4Z4 region of chromosome 4.

                          Product Name : EPI-321

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 18, 2023

                          Lead Product(s) : EPI-321

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Solve FSHD

                          Deal Size : Undisclosed

                          Deal Type : Financing

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                          08

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : SIG002 consists purified secretome free of tissue fragments and cells. SIG002 has demonstrated immunoregulatory properties relevant to address the degenerative inflammatory processes involved in osteoarthritis disease etiology.

                          Product Name : SIG002

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          January 31, 2023

                          Lead Product(s) : SIG002

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : SIS-101-ADO combines an siRNA that suppress expression of CLCN7, with SiSaf’s Bio-Courier® next generation silicon stabilized hybrid lipid nanoparticles technology of other RNA delivery technologies by merging LNP technology with inorganic bioabsorbab...

                          Product Name : SIS-101-ADO

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 12, 2022

                          Lead Product(s) : SIS-101-ADO

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : CSSi LifeSciences

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : FSD201 is a proprietary formulation of ultra-micronized palmitoylethanolamide (PEA). FSD Pharma’s successfully completed Phase 1 clinical trial showed FSD201 to be safe and well tolerated.

                          Product Name : FSD201

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 06, 2022

                          Lead Product(s) : Palmitoylethanolamide

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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