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Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0.26000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.26000000000000001,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Bioluminescence Ventures","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Bioluminescence Ventures"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"RCT1100","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"AskBio","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"SEL-399","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ AskBio","highestDevelopmentStatusID":"6","companyTruncated":"Selecta Biosciences \/ AskBio"},{"orgOrder":0,"company":"Azitra","sponsor":"Thinkequity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Probiotic","year":"2023","type":"Public Offering","leadProduct":"Strain of Staphylococcus Epidermidis","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Ointment","sponsorNew":"Azitra \/ Thinkequity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Thinkequity"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Probiotic","year":"2022","type":"Inapplicable","leadProduct":"SYNB1353","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"Synlogic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Probiotic","year":"2022","type":"Inapplicable","leadProduct":"SYNB1353","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Synlogic","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Synlogic \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Synlogic \/ Undisclosed"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"YOLT-203","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"YolTech Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"YolTech Therapeutics","sponsor":"Northwestern University","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"YOLT-203","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"YolTech Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"YolTech Therapeutics \/ Northwestern University","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Northwestern University"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"AUSTRALIA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neuren Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neuren Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Chromocell Corporation","sponsor":"Maxim Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Public Offering","leadProduct":"CC8464","moa":"Voltage-gated sodium channel alpha Nav1.7 (SCN9A)","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Chromocell Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Chromocell Corporation \/ Maxim Group LLC","highestDevelopmentStatusID":"6","companyTruncated":"Chromocell Corporation \/ Maxim Group LLC"}]

    Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR443809

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults

                            Details : SAR443809 is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            March 22, 2024

                            Lead Product(s) : SAR443809

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Jerry Vockley

                            Country arrow
                            BioJapan
                            Not Confirmed

                            Jerry Vockley

                            Country arrow
                            BioJapan
                            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) : Sodium Phenylbutyrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Zevra Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Open-Label Study of ACER-001 in Treatment of Combined D,L-2 Hydroxyglutaric Aciduria

                            Details : Sodium Phenylbutyrate is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of 2-Hydroxyglutaricaciduria.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 15, 2025

                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Zevra Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Kamari Pharma

                            Israel arrow
                            BioJapan
                            Not Confirmed

                            Kamari Pharma

                            Israel arrow
                            BioJapan
                            Not Confirmed
                            • fda
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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) : KM-023

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of KM-023 in Healthy Volunteers and Patients with Olmsted Syndrome.

                            Details : KM-023 is a drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Olmsted Syndrome.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            July 29, 2025

                            Lead Product(s) : KM-023

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Relief Therapeutics

                            Switzerland arrow
                            BioJapan
                            Not Confirmed

                            Relief Therapeutics

                            Switzerland arrow
                            BioJapan
                            Not Confirmed
                            • fda
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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) : Hypochlorous Acid

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Relief Therapeutics Receives FDA Rare Pediatric Disease Designation for RLF-TD011

                            Details : RLF-TD011 is a differentiated hypotonic, acid-oxidizing solution formulated with hypochlorous acid, offering strong antimicrobial and anti-inflammatory properties for epidermolysis bullosa.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 22, 2025

                            Lead Product(s) : Hypochlorous Acid

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            GRIN Therapeutics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            GRIN Therapeutics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed
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                            Lead Product(s) : Radiprodil

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Avance Clinical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Phase I Open-Label DDI Study of Radiprodil with Co-Administered Drugs

                            Details : Radiprodil is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Tuberous Sclerosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 16, 2025

                            Lead Product(s) : Radiprodil

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Avance Clinical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Quoin Pharmaceuticals

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            Quoin Pharmaceuticals

                            U.S.A arrow
                            BioJapan
                            Not Confirmed
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                            Lead Product(s) : QRX003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Quoin Pharma Posts Initial Positive QRX003 Data for Pediatric Skin Syndrome

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

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 14, 2025

                            Lead Product(s) : QRX003

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            GRIN Therapeutics

                            U.S.A arrow
                            BioJapan
                            Not Confirmed

                            GRIN Therapeutics

                            U.S.A arrow
                            BioJapan
                            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) : Radiprodil

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Avance Clinical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itrac...

                            Details : Radiprodil is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Tuberous Sclerosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 11, 2025

                            Lead Product(s) : Radiprodil

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Avance Clinical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Tianjin Huanhu Hospital

                            Country arrow
                            BioJapan
                            Not Confirmed

                            Tianjin Huanhu Hospital

                            Country arrow
                            BioJapan
                            Not Confirmed
                            • fda
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                            Lead Product(s) : Ucar T-Cell

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Shanghai Xiniao Biotech Co., Ltd.

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation A...

                            Details : Ucar T-Cell is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Neuromyelitis Optica.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            April 22, 2025

                            Lead Product(s) : Ucar T-Cell

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Shanghai Xiniao Biotech Co., Ltd.

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            F. Hoffmann-La Roche

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                            BioJapan
                            Not Confirmed

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            BioJapan
                            Not Confirmed
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                            Lead Product(s) : Rugonersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Oak Hill Bio

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Oak Hill Bio Licenses Phase 3 Angelman Drug Rights from Roche

                            Details : Under the licensing agreement, Oak will obtains the global rights of RO7248824 (rugonersen). Itis an ASO designed as a potential best-in-class treatment for individuals with Angelman syndrome.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            April 15, 2025

                            Lead Product(s) : Rugonersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Oak Hill Bio

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            10

                            Can-Fite BioPharma

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                            BioJapan
                            Not Confirmed

                            Can-Fite BioPharma

                            Israel arrow
                            BioJapan
                            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) : Piclidenoson

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Can-Fite Plans Phase II Trial of Piclidenoson for Lowe Syndrome

                            Details : CF101 (piclidenoson), a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule, orally bioavailable drug. It is being evaluated for lowe syndrome.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 19, 2025

                            Lead Product(s) : Piclidenoson

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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