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    [{"orgOrder":0,"company":"Pharvaris","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","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":"Santhera Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Omigapil","moa":"neuronal apoptosis inhibitors","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Santhera Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Santhera Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Nestle Health Sciences SA","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"CDX6114","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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nestle Health Sciences SA \/ Undisclosed"},{"orgOrder":0,"company":"Nestle Health Sciences SA","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Nestle Health Sciences SA \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2014","type":"Inapplicable","leadProduct":"AVXS-101","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Neurimmune","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Neurimmune \/ Undisclosed"},{"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"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & 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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Undisclosed","year":"2014","type":"Inapplicable","leadProduct":"RO6885247","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"F. Hoffmann-La Roche \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"F. Hoffmann-La Roche \/ Undisclosed"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Novartis Pharmaceuticals Corporation

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                            Novartis Pharmaceuticals Corporation

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                            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

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                            02

                            Vaderis Therapeutics

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                            Vaderis Therapeutics

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                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 18, 2024

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Relief Therapeutics

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                            Relief Therapeutics

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                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 04, 2024

                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Relief Therapeutics

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                            Relief Therapeutics

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                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 18, 2024

                            Lead Product(s) : Sapropterin Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Vaderis Therapeutics

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                            Vaderis Therapeutics

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                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 27, 2024

                            Lead Product(s) : VAD044

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Vaderis Therapeutics

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                            Vaderis Therapeutics

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                            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 : Miscellaneous

                            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

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                            07

                            F. Hoffmann-La Roche

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                            F. Hoffmann-La Roche

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                            Lead Product(s) : Crovalimab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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

                            Details : Crovalimab is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Anemia, Sickle Cell.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            June 03, 2021

                            Lead Product(s) : Crovalimab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            F. Hoffmann-La Roche

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                            F. Hoffmann-La Roche

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                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

                            Details : Risdiplam is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Muscular Atrophy, Spinal.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 22, 2021

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Pharvaris

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                            Pharvaris

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                            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 : Miscellaneous

                            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

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                            10

                            Pharvaris

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                            Pharvaris

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                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 13, 2020

                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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