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    [{"orgOrder":0,"company":"Novadip Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","upfrontCash":"Not Applicable","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","date":"March 2021","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","upfrontCash":"Not Applicable","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","date":"March 2022","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","upfrontCash":"Not Applicable","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","date":"May 2022","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","upfrontCash":"Undisclosed","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","date":"June 2022","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","upfrontCash":"Undisclosed","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","date":"June 2022","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","upfrontCash":"Not Applicable","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","date":"August 2022","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","upfrontCash":"Not Applicable","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","date":"August 2022","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","upfrontCash":"Undisclosed","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","date":"July 2023","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","upfrontCash":"Not Applicable","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","date":"October 2023","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

                            Quoin Pharmaceuticals

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                            Aquaexpo 2024
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                            Quoin Pharmaceuticals

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                            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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                            02

                            Rezolute

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                            Aquaexpo 2024
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                            Rezolute

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                            Aquaexpo 2024
                            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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                            03

                            Soligenix

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                            Aquaexpo 2024
                            Not Confirmed

                            Soligenix

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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 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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                            04

                            Soligenix

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                            Aquaexpo 2024
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                            Soligenix

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                            Aquaexpo 2024
                            Not Confirmed
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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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                            05

                            PaxMedica

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                            Aquaexpo 2024
                            Not Confirmed

                            PaxMedica

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                            Aquaexpo 2024
                            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) : 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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                            06

                            Caeregen Therapeutics

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                            Aquaexpo 2024
                            Not Confirmed

                            Caeregen Therapeutics

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                            Aquaexpo 2024
                            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) : 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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                            07

                            Benitec Biopharma

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                            Aquaexpo 2024
                            Not Confirmed

                            Benitec Biopharma

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                            Aquaexpo 2024
                            Not Confirmed
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                            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...

                            Brand Name : BB-301

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            June 26, 2023

                            Lead Product(s) : BB-301

                            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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                            08

                            Disc medicine

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                            Aquaexpo 2024
                            Not Confirmed

                            Disc medicine

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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : National Institutes of Health

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Disc Medicine Announces Collaboration with National Institutes of Health for Phase 2 Clinical Study of Bitoper...

                            Details : The collaboration will evaluate DISC-1459 (bitopertin), an orally administered inhibitor of GlyT1 that is designed to modulate heme biosynthesis, in a phase 2 clinical study of patients with Diamond-Blackfan anemia (DBA).

                            Brand Name : DISC-1459

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            March 21, 2023

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : National Institutes of Health

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            09

                            Benitec Biopharma

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                            Aquaexpo 2024
                            Not Confirmed

                            Benitec Biopharma

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                            Aquaexpo 2024
                            Not Confirmed
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                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : JMP Securities

                            Deal Size : $18.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Benitec Biopharma Inc. Announces Closing of $18 Million Underwritten Public Offering

                            Details : The Company intends to use the net proceeds from this financing for the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB301 treatment study.

                            Brand Name : BB-301

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 16, 2022

                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : JMP Securities

                            Deal Size : $18.0 million

                            Deal Type : Public Offering

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                            10

                            Benitec Biopharma

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                            Aquaexpo 2024
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                            Benitec Biopharma

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                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : JMP Securities

                            Deal Size : $18.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Benitec Biopharma Inc. Announces Pricing of $18 Million Underwritten Public Offering

                            Details : The company intends to use the proceeds for development of BB-301, including the natural history lead-in study and Phase 1b/2a BB-301 treatment study, for continued advancement of development activities, for general corporate purposes and for strategic g...

                            Brand Name : BB-301

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 12, 2022

                            Lead Product(s) : BB-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : JMP Securities

                            Deal Size : $18.0 million

                            Deal Type : Public Offering

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