menu
    • menu
    [{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTQ923","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Nestle Health Sciences SA","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"CDX-6114","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Nestle Health Sciences SA","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nestle Health Sciences SA \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Nestle Health Sciences SA \/ Inapplicable"},{"orgOrder":0,"company":"Neurimmune","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"NI006","moa":"Transthyretin (TTR)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Neurimmune","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurimmune \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Neurimmune \/ Inapplicable"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Series C Financing","leadProduct":"Deucrictibant","moa":"BDKRB2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pharvaris","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Undisclosed","sponsorNew":"Pharvaris \/ Viking Global Investors","highestDevelopmentStatusID":"6","companyTruncated":"Pharvaris \/ Viking Global Investors"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"EDK060","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

                            Loading...
                            refresh Filters
                            refresh Reset Filter
                            refresh Reset Filter
                            price-trend All Developments

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Novartis Pharmaceuticals Corporation

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Novartis Pharmaceuticals Corporation

                            Switzerland arrow
                            Boulder Peptide
                            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) : EDK060

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            First-in-Human Study of EDK060 Safety, Tolerability, & PK in Adults with CMT1A

                            Details : EDK060 is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Charcot-Marie-Tooth Disease.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            August 24, 2025

                            Lead Product(s) : EDK060

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Vaderis Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Vaderis Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            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) : VAD044

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Vaderis Gets FDA Fast Track for VAD044 in Hereditary Hemorrhagic Telangiectasia

                            Details : VAD044, a daily, oral allosteric AKT inhibitor, which has been investigated in a clinical proof of concept study in HHT patients.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 18, 2024

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Relief Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Relief Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            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) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Relief Completes Phase in RLF-OD032 Study; Topline Results Expected October 2024

                            Details : RLF-OD032, an innovative and highly concentrated liquid formulation of sapropterin dihydrochloride, is designed to lower blood phenylalanine in adult and pediatric PKU patients.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 18, 2024

                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Vaderis Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Vaderis Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            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) : VAD044

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Vaderis Announces Positive Clinical Proof-of-Concept Trial in HHT

                            Details : VAD044, a daily, oral allosteric AKT inhibitor, which has been investigated in a clinical proof of concept study in HHT patients.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 27, 2024

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Relief Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Relief Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            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) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Relief Therapeutics Reports Positive Results from RLF-OD032 Study For Phenylketonuria

                            Details : RLF-OD032 (sapropterin dihydrochloride) is a PAH agonist small molecule drug candidate which is being evaluated for the treatment of phenylketonuria.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 10, 2024

                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Vaderis Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Vaderis Therapeutics

                            Switzerland arrow
                            Boulder Peptide
                            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) : VAD044

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Medicxi

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Vaderis Therapeutics AG Emerges from Stealth and Announces Initiation of Clinical Proof-of-Concept Trial in HH...

                            Details : VAD044 is a once daily, orally administered, allosteric AKT inhibitor which has the potential to treat the underlying cause of Hereditary Haemorrhagic Telangiectasia (HHT), also known as Osler-Weber-Rendu Syndrome.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 22, 2022

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Medicxi

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            Boulder Peptide
                            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) : Crovalimab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Managemen...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            June 03, 2021

                            Lead Product(s) : Crovalimab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            Boulder Peptide
                            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) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 22, 2021

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Pharvaris

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Pharvaris

                            Switzerland arrow
                            Boulder Peptide
                            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) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Viking Global Investors

                            Deal Size : $80.0 million

                            Deal Type : Series C Financing

                            DEALS_DEV arrow-down

                            Pharvaris Announces $80 Million Series C Financing to Advance Novel Oral Bradykinin-B2-Receptor Antagonists fo...

                            Details : The proceeds from the Series C financing will fund the clinical advancement of Pharvaris’ pipeline of novel oral bradykinin-B2-receptor antagonists for the treatment of HAE, including both on-demand treatment and prophylactic prevention.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            November 18, 2020

                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Viking Global Investors

                            Deal Size : $80.0 million

                            Deal Type : Series C Financing

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            Pharvaris

                            Switzerland arrow
                            Boulder Peptide
                            Not Confirmed

                            Pharvaris

                            Switzerland arrow
                            Boulder Peptide
                            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) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pharvaris Presents Clinical Data at ACAAI 2020 Demonstrating Safety and Therapeutic Potential of Oral PHA121 f...

                            Details : The PK/PD profile suggests that rapid onset of action and prolonged efficacy with a single dose of PHA121 in the treatment of acute HAE attacks. Pharvaris is preparing to start an on-demand study of PHVS416, a soft capsule formulation of PHA121, for HAE ...

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 13, 2020

                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth