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Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Anavex Life Sciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Govorestat","moa":"Aldose reductase","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Omeros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Narsoplimab","moa":"MASP-2","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Omeros","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Omeros \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Omeros \/ Inapplicable"},{"orgOrder":0,"company":"Omeros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Narsoplimab","moa":"MASP-2","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Omeros","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Omeros \/ 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III","graph3":"Omeros","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Omeros \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Omeros \/ Inapplicable"},{"orgOrder":0,"company":"Rezolute","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Ersodetug","moa":"Insulin allosteric site receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rezolute \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Inapplicable"},{"orgOrder":0,"company":"Rezolute","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Ersodetug","moa":"Insulin allosteric site receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rezolute \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Exagamglogene Autotemcel","moa":"BCL11A","graph1":"Rare Diseases and Disorders","graph2":"Phase 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Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Savara","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Molgramostim","moa":"GM-CSF","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution for Nebulizer","sponsorNew":"Savara \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Inapplicable"},{"orgOrder":0,"company":"Saol therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Dichloroacetic Acid","moa":"PDK","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Saol therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Saol therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Saol therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Paradigm Therapeutics","sponsor":"Eshelman Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Financing","leadProduct":"Glyoxyldiureide","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Paradigm Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Topical Cream","sponsorNew":"Paradigm Therapeutics \/ Eshelman Ventures","highestDevelopmentStatusID":"10","companyTruncated":"Paradigm Therapeutics \/ Eshelman Ventures"},{"orgOrder":0,"company":"RHEACELL","sponsor":"AOP Health","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Cell and Gene therapy","year":"2025","type":"Agreement","leadProduct":"allo-APZ2-OTS","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"RHEACELL","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"RHEACELL \/ AOP Health","highestDevelopmentStatusID":"10","companyTruncated":"RHEACELL \/ AOP Health"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Protein","year":"2025","type":"Acquisition","leadProduct":"INZ-701","moa":"PPi\/Adenosine","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0.27000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Subcutaneous Injection","sponsorNew":"Inozyme Pharma \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ BioMarin Pharmaceutical"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            Inozyme Pharma

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                            Lead Product(s) : INZ-701

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : $270.0 million

                            Deal Type : Acquisition

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                            BioMarin Completes Acquisition of Inozyme Pharma for $270M

                            Details : The acquisition will strengthen BioMarin’s enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, for ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency.

                            Product Name : INZ-701

                            Product Type : Protein

                            Upfront Cash : $270.0 million

                            July 01, 2025

                            Lead Product(s) : INZ-701

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : $270.0 million

                            Deal Type : Acquisition

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                            RHEACELL

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                            Lead Product(s) : allo-APZ2-OTS

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : AOP Health

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            Rheacell and AOP Health Partner to Commercialize Cell Therapies in Europe

                            Details : Under the agreement, AOP will commercialize Rheacell’s two stem cell products, Ebesanar and Amesanar, in Europe, Turkey, Israel and the MENA region.

                            Product Name : Ebesanar

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 27, 2025

                            Lead Product(s) : allo-APZ2-OTS

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : AOP Health

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            Paradigm Therapeutics

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                            Lead Product(s) : Glyoxyldiureide

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Eshelman Ventures

                            Deal Size : $12.5 million

                            Deal Type : Financing

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                            Paradigm Therapeutics gets Eshelman Funding for SD-101 Topical EB Treatment

                            Details : The financing aims to advance the late-stage program Zorblisa (allantoin), which is being evaluated for the treatment of Epidermolysis Bullosa.

                            Product Name : Zorblisa

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            May 06, 2025

                            Lead Product(s) : Glyoxyldiureide

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Eshelman Ventures

                            Deal Size : $12.5 million

                            Deal Type : Financing

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                            04

                            Saol therapeutics

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                            Saol therapeutics

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                            Lead Product(s) : Dichloroacetic Acid

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Saol Therapeutics Announces Major Clinical Milestones Across Multiple Development Programs

                            Details : SL1009 is a sodium dichloroacetate, which inhibits PDK to stimulate residual PDC activity and ATP production by mitochondria. It is being investigated for pyruvate dehydrogenase complex deficiency.

                            Product Name : SL1009

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 17, 2025

                            Lead Product(s) : Dichloroacetic Acid

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Savara

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                            Savara

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                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Hercules Capital

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            Savara Enters Into Non-Dilutive Debt Financing for up to $200M with Hercules Capital

                            Details : The financing aims to fund the advance the clinical development of company's lead product Molbreevi (molgramostim). It is being evaluated for autoimmune pulmonary alveolar proteinosis.

                            Product Name : Molbreevi

                            Product Type : Protein

                            Upfront Cash : Undisclosed

                            March 26, 2025

                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Hercules Capital

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            06

                            Savara

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                            Savara

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                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Savara Submits BLA to FDA for MOLBREEVI for aPAP Treatment

                            Details : Molbreevi (molgramostim) is a GM-CSF agonist protein candidate. It is being evaluated for the treatment of autoimmune pulmonary alveolar proteinosis.

                            Product Name : Molbreevi

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            March 26, 2025

                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Omeros

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                            Omeros

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                            Lead Product(s) : Narsoplimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Omeros Announces Robust Results for Narsoplimab Expanded Access Program in TA-TMA

                            Details : OMS721 (Narsoplimab) is a MASP-2 inhibitor that targets the lectin pathway of complement for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy.

                            Product Name : OMS721

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            February 20, 2025

                            Lead Product(s) : Narsoplimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            InflaRx

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                            InflaRx

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                            Lead Product(s) : Ifx-1

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Guggenheim Securities

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            InflaRx Announces Closing of $30 Million Public Offering

                            Details : The net proceeds from the offering primarily to fund clinical development of its pipeline candidates, including Gohibic (vilobelimab), which is being evaluated for Pyoderma Gangrenosum.

                            Product Name : Gohibic

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            February 18, 2025

                            Lead Product(s) : Ifx-1

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Guggenheim Securities

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

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                            09

                            InflaRx

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                            InflaRx

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                            Lead Product(s) : Ifx-1

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Guggenheim Securities

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            InflaRx Prices $30M Public Offering of Shares & Pre-Funded Warrants

                            Details : The net proceeds from the offering will used to fund the clinical development of Gohibic (vilobelimab). It is being evaluated for the treatment of Pyoderma Gangraenosum.

                            Product Name : Gohibic

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            February 13, 2025

                            Lead Product(s) : Ifx-1

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Guggenheim Securities

                            Deal Size : $30.0 million

                            Deal Type : Public Offering

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                            10

                            INmune Bio

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                            INmune Bio

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                            Lead Product(s) : Umbilical Cord Derived Mesenchymal Stromal Cell

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            INmune Bio Plans FDA BLA Submission for CORDStrom in RDEB Treatment

                            Details : CORDStrom is an aseptic, allogeneic, pooled human umbilical cord -derived mesenchymal stromal cellsbeing investigated for recessive dystrophic epidermolysis bullosa.

                            Product Name : CORDStrom

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 10, 2025

                            Lead Product(s) : Umbilical Cord Derived Mesenchymal Stromal Cell

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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