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    [{"orgOrder":0,"company":"Novadip Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"NVD-003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Novadip Biosciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Novadip Biosciences \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Novadip Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"InnoCare Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Orelabrutinib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"InnoCare Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"InnoCare Pharma \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"InnoCare Pharma \/ Not Applicable"},{"orgOrder":0,"company":"DepYmed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"DPM-1001","moa":"PTP1B","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"DepYmed","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"DepYmed \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"DepYmed \/ Not Applicable"},{"orgOrder":0,"company":"Hong Kong Winhealth Pharma Group","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Licensing Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Hong Kong Winhealth Pharma Group","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Hong Kong Winhealth Pharma Group \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"5","companyTruncated":"Hong Kong Winhealth Pharma Group \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Cresence","sponsor":"Bioasis","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Acquisition","leadProduct":"CRES101","moa":"M2 polarization","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Cresence","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cresence \/ Bioasis","highestDevelopmentStatusID":"5","companyTruncated":"Cresence \/ Bioasis"},{"orgOrder":0,"company":"ImmunAbs","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"IM-101","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"ImmunAbs","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ImmunAbs \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"ImmunAbs \/ Not Applicable"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Equecabtagene Autoleucel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"IASO Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"IASO Bio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"IASO Bio \/ Not Applicable"},{"orgOrder":0,"company":"Caeregen Therapeutics","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Funding","leadProduct":"CTR-107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Caeregen Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Caeregen Therapeutics \/ National Institutes of Health","highestDevelopmentStatusID":"5","companyTruncated":"Caeregen Therapeutics \/ National Institutes of Health"},{"orgOrder":0,"company":"PaxMedica","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Suramin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"PaxMedica","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"PaxMedica \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"PaxMedica \/ Not Applicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            ImmuneSensor Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            ImmuneSensor Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : IMSB301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            ImmuneSensor Orphan and Pediatric Designations for IMSB301 in Aicardi Goutières Syndrome

                            Details : IMSB301 is a novel, orally available small molecule cGAS inhibitor drug candidate, that is being developed for the treatment of Aicardi Goutières Syndrome.

                            Brand Name : IMSB301

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            November 20, 2024

                            Lead Product(s) : IMSB301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            Quoin Pharmaceuticals

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Quoin Pharmaceuticals

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                            Medlab Asia & Asia Health
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : QRX003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Quoin Pharmaceuticals to Initiate Clinical Study for Peeling Skin Syndrome

                            Details : QRX003 is a topical lotion formulated with Invisicare®, a proprietary delivery technology, and contains a broad-spectrum serine protease inhibitor, being developed for Peeling skin syndrome.

                            Brand Name : QRX003

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            August 06, 2024

                            Lead Product(s) : QRX003

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            Rezolute

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Rezolute

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                            Medlab Asia & Asia Health
                            Not Confirmed
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                            Lead Product(s) : Ersodetug

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Rezolute Announces FDA Clearance for Phase 3 Study of RZ358 in Tumor Hyperinsulinism

                            Details : RZ358, which is an insulin receptor allosteric site activator. Currently, it is being evaluated for the treatment of hypoglycemia in patients with tumor hyperinsulinism.

                            Brand Name : RZ358

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            August 05, 2024

                            Lead Product(s) : Ersodetug

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            Soligenix

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Soligenix

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Dusquetide

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            FDA Grants Fast Track Designation to Dusquetide for Oral Lesions in Behcet's Disease

                            Details : SGX945 (dusquetide) is an innate defense regulator (p62 protein modulator), a new class of short, synthetic peptides. It is being evaluated for the treatment of lesions of Behçet's Disease

                            Brand Name : SGX945

                            Molecule Type : Peptide

                            Upfront Cash : Not Applicable

                            January 08, 2024

                            Lead Product(s) : Dusquetide

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Soligenix

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Soligenix

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : Dusquetide

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            FDA Grants Fast Track Designation for Dusquetide for Oral Lesions of Behçet's Disease

                            Details : SGX945 (dusquetide) is an innate defense regulator (IDR), a new class of short, synthetic peptides. It is being evaluated in IND enabling studies for the treatment of oral lesions of Behçet's Disease.

                            Brand Name : SGX945

                            Molecule Type : Peptide

                            Upfront Cash : Not Applicable

                            January 08, 2024

                            Lead Product(s) : Dusquetide

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            PaxMedica

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            PaxMedica

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            • EDQM
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Suramin

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PaxMedica Holds Important FDA Type-B Meeting to Review PAX-101 Pivotal Trial Data

                            Details : PAX-101 (suramin sodium) is a intravenous formulation for Autism Spectrum Disorder. Suramin is a broadly acting anti-purinergic therapy and has reported positive results from a dose range study.

                            Brand Name : PAX-101

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 27, 2023

                            Lead Product(s) : Suramin

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Caeregen Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Caeregen Therapeutics

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : CTR-107

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : National Institutes of Health

                            Deal Size : $1.4 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Caeregen Therapeutics Receives $1.4 Million Phase 2 SBIR Grant from NIH/NEI to Advance Development of Regenera...

                            Details : The funding will be used to advance the development of Noregen (CTR-107), a novel regenerative therapeutic for the treatment of retinal-related vision loss due to a wide range of inherited and age-related diseases.

                            Brand Name : Noregen

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            July 17, 2023

                            Lead Product(s) : CTR-107

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : National Institutes of Health

                            Deal Size : $1.4 million

                            Deal Type : Funding

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                            08

                            Benitec Biopharma

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed

                            Benitec Biopharma

                            U.S.A arrow
                            Medlab Asia & Asia Health
                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Benitec Biopharma Receives FDA Clearance of the IND for BB-301 for the Treatment of Oculopharyngeal Muscular D...

                            Details : BB-301 is a novel, modified AAV9 capsid expressing bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 and two small inhibitory RNAs against mutant PABPN1 is currently in development for Oculopharyngeal...