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Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-006","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"X Vivo Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"Genethon","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"X Vivo Gene Therapy","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Undisclosed"},{"orgOrder":0,"company":"Chigenovo","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"ZVS203e","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Chigenovo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chigenovo \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Chigenovo \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2018","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Schmidhofer, Mark, MD","sponsor":"Savara | Cystic Fibrosis Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Sodium Nitrite","moa":"Vasodilator","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Schmidhofer, Mark, MD","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Schmidhofer, Mark, MD \/ Savara | Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Schmidhofer, Mark, MD \/ Savara | Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"Gabrail Cancer Center Research","sponsor":"Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Amino Acid","year":"2015","type":"Inapplicable","leadProduct":"L-Arginine","moa":"Vasopressin receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Gabrail Cancer Center Research","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health","highestDevelopmentStatusID":"7","companyTruncated":"Gabrail Cancer Center Research \/ Children's Healthcare of Atlanta | National Center for Complementary and Integrative Health"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"HMB-002","moa":"VMF\/Factor VII level","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"HMB-002","moa":"VMF\/Factor VII level","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hemab Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sapience Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"Irinotecan","moa":"||Wnt\/beta-catenin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sapience Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sapience Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sapience Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Agtc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"AGTC-501","moa":"X-linked retinitis pigmentosa GTPase regulator (RPGR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"BIIB112","moa":"X-linked retinitis pigmentosa GTPase regulator (RPGR)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Leiden University Medical Centre","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mustang Bio \/ Leiden University Medical Centre","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Leiden University Medical Centre"},{"orgOrder":0,"company":"Ensoma","sponsor":"Gilead Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"EN-374","moa":"CYBB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ensoma","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"Ensoma \/ Gilead Sciences","highestDevelopmentStatusID":"7","companyTruncated":"Ensoma \/ Gilead Sciences"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"AAVB-039","moa":"ABCA4","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AAVantgarde Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Undisclosed"},{"orgOrder":0,"company":"GeneCradle Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"GC310","moa":"ATP7B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneCradle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneCradle Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GeneCradle Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alesta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"ALE1","moa":"PPi","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Alesta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alesta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Alesta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Financing","leadProduct":"Antimony pentoxide","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Hercules Capital","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Hercules Capital"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell & Gene Therapy","year":"2025","type":"Public Offering","leadProduct":"Antimony pentoxide","moa":"Huntingtin (HTT)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0.34999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Financing","leadProduct":"RCT2100","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"ReCode Therapeutics \/ Cystic Fibrosis Foundation"}]

    Find Drugs for Genetic Disease in Phase I/ Phase II 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) : SAR446268

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-m...

                            Details : SAR446268 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Myotonic Dystrophy.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            February 25, 2025

                            Lead Product(s) : SAR446268

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            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) : Etentamig

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Change in Disease Activity and Adverse Events (AE)s in Adult Participants With Immunoglobuli...

                            Details : Etentamig is a Antibody drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            December 06, 2023

                            Lead Product(s) : Etentamig

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : SAR444836

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous ...

                            Details : SAR444836 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Phenylketonurias.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            August 02, 2023

                            Lead Product(s) : SAR444836

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            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) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

                            Details : Fosigotifator is a Other Small Molecule drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Leukoencephalopathies.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 07, 2023

                            Lead Product(s) : Fosigotifator

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Translate Bio Provides MRT5005 Program Updates

                            Details : Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            06

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis

                            Details : First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : MRT5201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Defici...

                            Details : MRT5201 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Ornithine Carbamoyltransferase Deficiency Disease.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            December 06, 2018

                            Lead Product(s) : MRT5201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibr...

                            Details : MRT5005 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            December 15, 2017

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : Recombinant Antihemophilic Factor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulatio...

                            Details : Recombinant Antihemophilic Factor is a Protein drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            July 02, 2017

                            Lead Product(s) : Recombinant Antihemophilic Factor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            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) : RST-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa

                            Details : RST-001 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Retinitis Pigmentosa.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 22, 2015

                            Lead Product(s) : RST-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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