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Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Epicrispr Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"EPI-321","moa":"DUX4 expression","graph1":"Musculoskeletal","graph2":"Phase I\/ Phase II","graph3":"Epicrispr Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Epicrispr Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Epicrispr Biotechnologies \/ Inapplicable"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : The partnership aims to advance the clinical development of EPI-321, which is being evaluated in ther early-stage clinical trial studies for the treatment of FSHD.

                          Product Name : EPI-321

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 23, 2025

                          Lead Product(s) : EPI-321

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Springbok Analytics

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          02

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Allocetra-OTS, fundamental approach centered on restoring macrophage homeostasis, currently being investigated for knee osteoarthritis.

                          Product Name : Allocetra-OTS

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 21, 2025

                          Lead Product(s) : Allocetra-OTS

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : EPI-321, a first-in-class epigenetic therapy for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disease.

                          Product Name : EPI-321

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 03, 2025

                          Lead Product(s) : EPI-321

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          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 knee osteoarthritis.

                          Product Name : Allocetra

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 03, 2025

                          Lead Product(s) : Allocetra

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : NVD-003 is an autologous cell-based therapy which is is made from a patient's own adipose stem cells. It is being evaluated for the treatment of congenital pseudarthrosis of tibia.

                          Product Name : NVD-003

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 28, 2025

                          Lead Product(s) : NVD-003

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : The financing aims to advance the clinical development of IMM01-STEM, which is being evaluated for the treatment of age-related muscle atrophy.

                          Product Name : IMM01-STEM

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 11, 2025

                          Lead Product(s) : IMM01-STEM

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Remiges Ventures

                          Deal Size : $25.0 million

                          Deal Type : Series A Financing

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                          07

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          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 knee osteoarthritis.

                          Product Name : Allocetra-OTS

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 11, 2024

                          Lead Product(s) : Allocetra-OTS

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          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 : Allocetra

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 11, 2024

                          Lead Product(s) : Allocetra

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Under the license agreement, Sarepta will gain exclusive global rights to several clinical and preclinical programs, including ARO-DUX4, which is being developed as a treatment for FSHD.

                          Product Name : ARO-DUX4

                          Product Type : Oligonucleotide

                          Upfront Cash : $825.0 million

                          November 26, 2024

                          Lead Product(s) : ARO-DUX4

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Sarepta Therapeutics

                          Deal Size : $11,375.0 million

                          Deal Type : Licensing Agreement

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                          10

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : NVDX3 is in clinical development for common orthopedic conditions. Currently, it is being evaluated in the early-stage clinical trial studies for cervical spine fusion.

                          Product Name : NVD-X3

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 20, 2024

                          Lead Product(s) : NVD-X3

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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