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Ventures"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Agreement","leadProduct":"Prademagene Zamikeracel","moa":"Type VII collagen","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Abeona Therapeutics","amount2":0.16,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Topical Sheet","sponsorNew":"Abeona Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Abeona Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Azafaros","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2025","type":"Series B Financing","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Azafaros","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Jeito Capital","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Jeito Capital"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Quoin Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Miglustat","moa":"Glucosylceramide synthase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"MavriX Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"MVX-220","moa":"UBE3A gene","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"MavriX Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intra-cisterna magna Injection","sponsorNew":"MavriX Bio \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"MavriX Bio \/ Inapplicable"},{"orgOrder":0,"company":"RevolKa","sponsor":"D3 LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Protein","year":"2025","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"RevolKa","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"RevolKa \/ D3 LLC","highestDevelopmentStatusID":"3","companyTruncated":"RevolKa \/ D3 LLC"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Ruxoprubart","moa":"CFB","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"NovelMed Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Hypochlorous Acid","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Spray","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"AAV9","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"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":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"AAV9","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Kamari Pharma","sponsor":"BRM Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Other Small Molecule","year":"2025","type":"Series A Financing","leadProduct":"KM023","moa":"TRPV3","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Kamari Pharma","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Oral","sponsorNew":"Kamari Pharma \/ BRM Group","highestDevelopmentStatusID":"4","companyTruncated":"Kamari Pharma \/ BRM Group"},{"orgOrder":0,"company":"Actio Biosciences","sponsor":"Regeneron Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Series B Financing","leadProduct":"ABS-1230","moa":"KCNT1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Actio Biosciences","amount2":0.070000000000000007,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Oral","sponsorNew":"Actio Biosciences \/ Regeneron Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Actio Biosciences \/ Regeneron Ventures"}]

    Find Clinical Drug Pipelines for Rare Diseases and Disorders

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                            01

                            Breckenridge Pharmaceutical

                            U.S.A
                            Indo Livestock Expo
                            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) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Breckenridge Gains FDA Nod for Everolimus, Afinitor Disperz Generic

                            Details : Afinitor Disperz (everolimus) is an mTOR inhibitor small molecule drug candidate, which is currently being evaluated for the treatment of tuberous sclerosis complex.

                            Product Name : Afinitor Disperz-Generic

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 29, 2025

                            Lead Product(s) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Breckenridge Pharmaceutical

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                            02

                            Orion Corporation

                            Finland
                            Indo Livestock Expo
                            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) : Ganaxolone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            European Commission Approval of Ztalmy (ganaxolone) for the Adjunctive Treatment of Epileptic Seizures Associa...

                            Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

                            Product Name : Ztalmy

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 31, 2023

                            Lead Product(s) : Ganaxolone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Fermion Orion Company Banner

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                            03

                            Zenara Pharma

                            India
                            Indo Livestock Expo
                            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) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biophore, Its Subsidiary Zenara Pharma, Receive First Approval for Cannabidiol in India

                            Details : Cannabinoid oral solution has received approval for the treatment of several neurological disorders. It functions by stimulating two receptors, cannabinoid receptor type 1 and type 2, within the endocannabinoid system, maintaining body's homeostatis.

                            Product Name : Undisclosed

                            Product Type : Controlled Substance

                            Upfront Cash : Inapplicable

                            May 15, 2023

                            Lead Product(s) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Biophore CB

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                            04

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Olipudase alfa Shown to Provide Sustained Improvement Across Multiple Clinical Manifestations of ASMD

                            Details : Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            September 02, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            05

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme TM (Olipudase Alfa-rpcp) Approved by FDA as First Disease-specific Treatment for ASMD (Non-CNS Manif...

                            Details : Xenpozyme, (olipudase Alfa-rpcp) hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase, an enzyme that allows for the breakdown of the lipid sphingomyelin.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            August 31, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            06

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme® (olipudase alfa) Approved by European Commission as First and Only treatment for ASMD

                            Details : Xenpozyme (Olipudase alfa), is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 28, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            07

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            CHMP Recommends Approval of Xenpozyme® (Olipudase Alfa), the First and Only Treatment for ASMD

                            Details : Recommendation based on positive results from two clinical trials in which Xenpozyme (olipudase alfa) provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            May 19, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme® (olipudase alfa), Approved in Japan, First and Only Approved Therapy Indicated to Treat Acid Sphin...

                            Details : Approval based on positive results of Xenpozyme® (olipudase alfa) from two separate clinical trials in children and adults demonstrating improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            March 28, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            09

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            EMA Accepts Regulatory Submission for Olipudase Alfa, the First Potential Therapy for ASMD

                            Details : Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 12, 2021

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            10

                            Axplora

                            Germany
                            PREP
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothAxplora is your partner of choice for complex APIs.

                            Lead Product(s) : OTOF-GT,Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Preclinical

                            Sponsor : Sensorion

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement

                            Details : Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

                            Product Name : OTOF-GT

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            October 27, 2020

                            Lead Product(s) : OTOF-GT,Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Preclinical

                            Sponsor : Sensorion

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            Axplora CB

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