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    [{"orgOrder":0,"company":"Calcilytix Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Encaleret","moa":"calcium receptor antagonists","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calcilytix Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Calcilytix Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Calcilytix Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"medac","sponsor":"Celerion | Venn Life Sciences | Syneos Health","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cytotoxic Drug","year":"2015","type":"Inapplicable","leadProduct":"Treosulfan","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"medac","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"medac \/ Celerion | Venn Life Sciences | Syneos Health","highestDevelopmentStatusID":"8","companyTruncated":"medac \/ Celerion | Venn Life Sciences | Syneos Health"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"BI1291583","moa":"DPP-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"BI1265162","moa":"ENaC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Merck Group","sponsor":"Merck Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Hormone","year":"2010","type":"Inapplicable","leadProduct":"Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Merck Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Merck Group \/ Merck Group","highestDevelopmentStatusID":"8","companyTruncated":"Merck Group \/ Merck Group"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Riociguat","moa":"Soluble guanylate cyclase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Merck & Co","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Merck & Co"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2018","type":"Inapplicable","leadProduct":"Befovacimab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"BAY 3401016","moa":"SEMA3A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Romain Lazor","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Nintedanib Esylate","moa":"Platelet-derived growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Romain Lazor","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Romain Lazor"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            medac

                            Germany
                            CPhI India
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Boothmedac CDMO: Tailored CDMO services for injectables in syringes, cartridges & vials – aseptic, reliable, flexible and a solution ahead.

                            Lead Product(s) : Treosulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Celerion | Venn Life Sciences | Syneos Health

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases

                            Details : Treosulfan is a Cytotoxic Drug drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Immunologic Deficiency Syndromes.

                            Product Name : Undisclosed

                            Product Type : Cytotoxic Drug

                            Upfront Cash : Inapplicable

                            January 29, 2015

                            Lead Product(s) : Treosulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Celerion | Venn Life Sciences | Syneos Health

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Bayer AG

                            Germany arrow
                            CPhI India
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                            Bayer AG

                            Germany arrow
                            CPhI India
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                            • 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) : BAY 3401016

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BAY3401016; Biomarker Study Alport

                            Details : BAY 3401016 is a Antibody, Unconjugated drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Nephritis, Hereditary.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            October 08, 2025

                            Lead Product(s) : BAY 3401016

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Boehringer Ingelheim GmbH

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                            Boehringer Ingelheim GmbH

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

                            Lead Product(s) : BI1291583

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairaflyâ„...

                            Details : BI1291583 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 19, 2023

                            Lead Product(s) : BI1291583

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Boehringer Ingelheim GmbH

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                            CPhI India
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                            Boehringer Ingelheim GmbH

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                            CPhI India
                            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) : Nintedanib Esylate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Romain Lazor

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients

                            Details : Nintedanib is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Telangiectasia, Hereditary Hemorrhagic.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 26, 2021

                            Lead Product(s) : Nintedanib Esylate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Romain Lazor

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Calcilytix Therapeutics

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                            CPhI India
                            Not Confirmed

                            Calcilytix Therapeutics

                            Germany arrow
                            CPhI India
                            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) : Encaleret

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BridgeBio Pharma’s Calcilytix Therapeutics Initiates Phase 2 Study Of Encaleret For Autosomal Dominant Hypoc...

                            Details : The Phase 2 trial will enroll up to 16 individuals with ADH1 in an open-label, dose-ranging study to evaluate the safety, tolerability, pharmacodynamics, pharmacokinetics, and efficacy of single and multiple doses of encaleret.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 21, 2020

                            Lead Product(s) : Encaleret

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Boehringer Ingelheim GmbH

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                            Boehringer Ingelheim GmbH

                            Germany arrow
                            CPhI India
                            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) : BI1265162

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the ...

                            Details : BI1265162 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 16, 2019

                            Lead Product(s) : BI1265162

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Bayer AG

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                            Bayer AG

                            Germany arrow
                            CPhI India
                            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) : Befovacimab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

                            Details : Befovacimab is a Antibody drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            July 24, 2018

                            Lead Product(s) : Befovacimab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Bayer AG

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                            CPhI India
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                            Bayer AG

                            Germany arrow
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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) : Riociguat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Merck & Co

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients

                            Details : Adempas (Riociguat) is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Adempas

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 23, 2014

                            Lead Product(s) : Riociguat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Merck & Co

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Merck Group

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                            CPhI India
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                            Merck Group

                            Germany arrow
                            CPhI India
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                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Recombinant Human Growth Hormone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Merck Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Chi...

                            Details : Recombinant Human Growth Hormone is a Hormone drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hypochondroplasia.

                            Product Name : Undisclosed

                            Product Type : Hormone

                            Upfront Cash : Inapplicable

                            April 27, 2010

                            Lead Product(s) : Recombinant Human Growth Hormone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Merck Group

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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