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    [{"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":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pharvaris","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"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":"PAH gene","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":"PAH gene","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":"Vaderis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"VAD044","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Vaderis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Vaderis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"VAD044","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Vaderis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Vaderis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Medicxi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"VAD044","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vaderis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Vaderis Therapeutics \/ Medicxi","highestDevelopmentStatusID":"6","companyTruncated":"Vaderis Therapeutics \/ Medicxi"},{"orgOrder":0,"company":"Neurimmune","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"NI006","moa":"ATTR","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"}]

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                            01

                            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 : 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 : VAD044

                            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

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                            02

                            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 : 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 : RLF-OD032

                            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

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                            03

                            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 : 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 : VAD044

                            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

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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 : 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 : RLF-OD032

                            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

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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 : 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 : VAD044

                            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

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                            06

                            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 : PHVS416

                            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

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                            07

                            Pharvaris

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                            Pharvaris

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                            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 : PHVS416

                            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

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                            08

                            Novartis Pharmaceuticals Corporation

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                            EPS
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                            Novartis Pharmaceuticals Corporation

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                            EPS
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                            Lead Product(s) : Onasemnogene Abeparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

                            Details : Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (IT) formulation in older patients with SMA to further support registration.

                            Product Name : Zolgensma

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            September 23, 2020

                            Lead Product(s) : Onasemnogene Abeparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Pharvaris

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                            EPS
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                            Pharvaris

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                            EPS
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                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PHA121 Shows Clinically Relevant Activity in a Translational in Vivo Model

                            Details : The objective of the study was to investigate the ability of PHA121 to attenuate blood-pressure changes induced by bradykinin injection.

                            Product Name : PHVS416

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 04, 2020

                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Pharvaris

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                            Pharvaris

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                            EPS
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                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Preclinical Characterization of Novel B2-Receptor Antagonists Published in Frontiers in Pharmacology

                            Details : The data set found in the publication demonstrates the preclinical profile of the new class of molecules relative to icatibant, an injectable B2-receptor antagonist used as the leading therapy for on-demand treatment of HAE.

                            Product Name : PHVS416

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 30, 2020

                            Lead Product(s) : Deucrictibant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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