Cystic Fibrosis – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Cystic Fibrosis – Pipeline Review, H2 2019’, provides an overview of the Cystic Fibrosis pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Cystic Fibrosis, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Cystic Fibrosis and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.”

Scope


– The report provides a snapshot of the global therapeutic landscape of Cystic Fibrosis

– The report reviews pipeline therapeutics for Cystic Fibrosis by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Cystic Fibrosis therapeutics and enlists all their major and minor projects

– The report assesses Cystic Fibrosis therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Cystic Fibrosis”

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Cystic Fibrosis

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Cystic Fibrosis pipeline depth and focus of Indication therapeutics

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

“4D Molecular Therapeutics Inc

AbbVie Inc

Abeona Therapeutics Inc

Agile Sciences Inc

AGILeBiotics BV

Alaxia SAS

AlgiPharma AS

Antabio SAS

Arch Biopartners Inc

Arcturus Therapeutics Ltd

Armata Pharmaceuticals Inc

Arrevus Inc

Arrowhead Pharmaceuticals Inc

AstraZeneca Plc

Avidin Ltd

BioAegis Therapeutics Inc

Biolytx Pharmaceuticals Corp

Boehringer Ingelheim International GmbH

Breathe Easy Therapeutics Ltd

Calista Therapeutics Inc

Calithera Biosciences Inc

Catabasis Pharmaceuticals Inc

Celtaxsys Inc

Chiesi Farmaceutici SpA

Chrysalis Pharma SAS

Cilian AG

Corbus Pharmaceuticals Inc

CRISPR Therapeutics AG

CrowdOut Therapeutics

Cyclacel Pharmaceuticals Inc

Cyclenium Pharma Inc

Daiichi Sankyo Co Ltd

Destiny Pharma Plc

DiscoveryBiomed Inc

Druggability Technologies Holdings Ltd

Editas Medicine Inc

Eloxx Pharmaceuticals Inc

EmphyCorp Inc

EnBiotix Inc

Enterprise Therapeutics Ltd

Entrinsic Health Solutions, Inc.

Enzyvant Sciences Ltd

Errant Gene Therapeutics LLC

Evaxion Biotech ApS

Evotec SE

Exotect LLC

Feldan Therapeutics Inc

Galapagos NV

Generation Bio Corp

Genzyme Corp

Grupo Praxis Pharmaceutical SA

Helperby Therapeutics Group Ltd

Horizon Therapeutics PLC

Hypo-Stream Ltd

Icagen Inc

ID Pharma Co Ltd

Idorsia Pharmaceutical Ltd

Immun System IMS AB

Invion Ltd

Ionis Pharmaceuticals Inc

Kamada Ltd

Kither Biotech Srl

Kyorin Pharmaceutical Co Ltd

Lakewood-Amedex Inc

Lamellar Biomedical Ltd

Laurent Pharmaceuticals Inc

Lexicon Pharmaceuticals Inc

MannKind Corp

Mariposa Health Ltd

Microbion Corp

Mucokinetica Ltd

Nanogenics Ltd

Neupharma Srl

New Amsterdam Sciences Inc

Novabiotics Ltd

Novartis AG

Novoteris LLC

Omnispirant Ltd

Onspira Therapeutics Inc

Orphomed Inc

OrPro Therapeutics Inc

Oryn Therapeutics

Paranta Biosciences Ltd

Pharmaero ApS

Pharmaxis Ltd

PhaseBio Pharmaceuticals Inc

Polyphor AG

Progenra Inc

ProQR Therapeutics NV

Protalix BioTherapeutics Inc

Proteostasis Therapeutics Inc

Pulmatrix Inc

Rare Partners Srl

Recode Therapeutics Inc

Renovion Inc

ReveraGen BioPharma Inc

Riptide Bioscience Inc

Santhera Pharmaceuticals Holding AG

SciBac Inc

Sequoia Sciences Inc

Silurian Pharmaceuticals Inc

SolAeroMed Inc

Spirovant Sciences Inc

Spyryx Biosciences Inc

Synedgen Inc

Synovo GmbH

Synspira LLC

Takeda Pharmaceutical Co Ltd

TGV-Inhalonix Inc

Translate Bio Inc

Trucode Gene Repair Inc

Vast Therapeutics Inc

Vectura Group Plc

Verona Pharma Plc

Vertex Pharmaceuticals Inc

Vironika LLC

Zambon Co SpA

Zata Pharmaceuticals Inc”

Table of Contents

Table of Contents

Table of Contents 2

Introduction 6

Cystic Fibrosis Overview 7

Cystic Fibrosis Therapeutics Development 8

Cystic Fibrosis Therapeutics Assessment 29

Cystic Fibrosis Companies Involved in Therapeutics Development 41

Cystic Fibrosis Drug Profiles 76

Cystic Fibrosis Dormant Projects 395

Cystic Fibrosis Discontinued Products 403

Cystic Fibrosis Product Development Milestones 404

Appendix 417

List of Tables

“List of Tables

Number of Products under Development for Cystic Fibrosis, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..2), H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..3), H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..4), H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..5), H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Products under Development by Companies, H2 2019 (Contd..2), H2 2019

Products under Development by Companies, H2 2019 (Contd..3), H2 2019

Products under Development by Companies, H2 2019 (Contd..4), H2 2019

Products under Development by Companies, H2 2019 (Contd..5), H2 2019

Products under Development by Companies, H2 2019 (Contd..6), H2 2019

Products under Development by Companies, H2 2019 (Contd..7), H2 2019

Products under Development by Companies, H2 2019 (Contd..8), H2 2019

Products under Development by Companies, H2 2019 (Contd..9), H2 2019

Products under Development by Universities/Institutes, H2 2019

Products under Development by Universities/Institutes, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Target, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Target, H2 2019 (Contd..2), H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019 (Contd..2), H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Cystic Fibrosis – Pipeline by 4D Molecular Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by AbbVie Inc, H2 2019

Cystic Fibrosis – Pipeline by Abeona Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Agile Sciences Inc, H2 2019

Cystic Fibrosis – Pipeline by AGILeBiotics BV, H2 2019

Cystic Fibrosis – Pipeline by Alaxia SAS, H2 2019

Cystic Fibrosis – Pipeline by AlgiPharma AS, H2 2019

Cystic Fibrosis – Pipeline by Antabio SAS, H2 2019

Cystic Fibrosis – Pipeline by Arch Biopartners Inc, H2 2019

Cystic Fibrosis – Pipeline by Arcturus Therapeutics Ltd, H2 2019

Cystic Fibrosis – Pipeline by Armata Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Arrevus Inc, H2 2019

Cystic Fibrosis – Pipeline by Arrowhead Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by AstraZeneca Plc, H2 2019

Cystic Fibrosis – Pipeline by Avidin Ltd, H2 2019

Cystic Fibrosis – Pipeline by BioAegis Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Biolytx Pharmaceuticals Corp, H2 2019

Cystic Fibrosis – Pipeline by Boehringer Ingelheim International GmbH, H2 2019

Cystic Fibrosis – Pipeline by Breathe Easy Therapeutics Ltd, H2 2019

Cystic Fibrosis – Pipeline by Calista Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Calithera Biosciences Inc, H2 2019

Cystic Fibrosis – Pipeline by Catabasis Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Celtaxsys Inc, H2 2019

Cystic Fibrosis – Pipeline by Chiesi Farmaceutici SpA, H2 2019

Cystic Fibrosis – Pipeline by Chrysalis Pharma SAS, H2 2019

Cystic Fibrosis – Pipeline by Cilian AG, H2 2019

Cystic Fibrosis – Pipeline by Corbus Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by CRISPR Therapeutics AG, H2 2019

Cystic Fibrosis – Pipeline by CrowdOut Therapeutics, H2 2019

Cystic Fibrosis – Pipeline by Cyclacel Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Cyclenium Pharma Inc, H2 2019

Cystic Fibrosis – Pipeline by Daiichi Sankyo Co Ltd, H2 2019

Cystic Fibrosis – Pipeline by Destiny Pharma Plc, H2 2019

Cystic Fibrosis – Pipeline by DiscoveryBiomed Inc, H2 2019

Cystic Fibrosis – Pipeline by Druggability Technologies Holdings Ltd, H2 2019

Cystic Fibrosis – Pipeline by Editas Medicine Inc, H2 2019

Cystic Fibrosis – Pipeline by Eloxx Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by EmphyCorp Inc, H2 2019

Cystic Fibrosis – Pipeline by EnBiotix Inc, H2 2019

Cystic Fibrosis – Pipeline by Enterprise Therapeutics Ltd, H2 2019

Cystic Fibrosis – Pipeline by Entrinsic Health Solutions, Inc., H2 2019

Cystic Fibrosis – Pipeline by Enzyvant Sciences Ltd, H2 2019

Cystic Fibrosis – Pipeline by Errant Gene Therapeutics LLC, H2 2019

Cystic Fibrosis – Pipeline by Evaxion Biotech ApS, H2 2019

Cystic Fibrosis – Pipeline by Evotec SE, H2 2019

Cystic Fibrosis – Pipeline by Exotect LLC, H2 2019

Cystic Fibrosis – Pipeline by Feldan Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Galapagos NV, H2 2019

Cystic Fibrosis – Pipeline by Generation Bio Corp, H2 2019

Cystic Fibrosis – Pipeline by Genzyme Corp, H2 2019

Cystic Fibrosis – Pipeline by Grupo Praxis Pharmaceutical SA, H2 2019

Cystic Fibrosis – Pipeline by Helperby Therapeutics Group Ltd, H2 2019

Cystic Fibrosis – Pipeline by Horizon Therapeutics PLC, H2 2019

Cystic Fibrosis – Pipeline by Hypo-Stream Ltd, H2 2019

Cystic Fibrosis – Pipeline by Icagen Inc, H2 2019

Cystic Fibrosis – Pipeline by ID Pharma Co Ltd, H2 2019

Cystic Fibrosis – Pipeline by Idorsia Pharmaceutical Ltd, H2 2019

Cystic Fibrosis – Pipeline by Immun System IMS AB, H2 2019

Cystic Fibrosis – Pipeline by Invion Ltd, H2 2019

Cystic Fibrosis – Pipeline by Ionis Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Kamada Ltd, H2 2019

Cystic Fibrosis – Pipeline by Kither Biotech Srl, H2 2019

Cystic Fibrosis – Pipeline by Kyorin Pharmaceutical Co Ltd, H2 2019

Cystic Fibrosis – Pipeline by Lakewood-Amedex Inc, H2 2019

Cystic Fibrosis – Pipeline by Lamellar Biomedical Ltd, H2 2019

Cystic Fibrosis – Pipeline by Laurent Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Lexicon Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by MannKind Corp, H2 2019

Cystic Fibrosis – Pipeline by Mariposa Health Ltd, H2 2019

Cystic Fibrosis – Pipeline by Microbion Corp, H2 2019

Cystic Fibrosis – Pipeline by Mucokinetica Ltd, H2 2019

Cystic Fibrosis – Pipeline by Nanogenics Ltd, H2 2019

Cystic Fibrosis – Pipeline by Neupharma Srl, H2 2019

Cystic Fibrosis – Pipeline by New Amsterdam Sciences Inc, H2 2019

Cystic Fibrosis – Pipeline by Novabiotics Ltd, H2 2019

Cystic Fibrosis – Pipeline by Novartis AG, H2 2019

Cystic Fibrosis – Pipeline by Novoteris LLC, H2 2019

Cystic Fibrosis – Pipeline by Omnispirant Ltd, H2 2019

Cystic Fibrosis – Pipeline by Onspira Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Orphomed Inc, H2 2019

Cystic Fibrosis – Pipeline by OrPro Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Oryn Therapeutics, H2 2019

Cystic Fibrosis – Pipeline by Paranta Biosciences Ltd, H2 2019

Cystic Fibrosis – Pipeline by Pharmaero ApS, H2 2019

Cystic Fibrosis – Pipeline by Pharmaxis Ltd, H2 2019

Cystic Fibrosis – Pipeline by PhaseBio Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Polyphor AG, H2 2019

Cystic Fibrosis – Pipeline by Progenra Inc, H2 2019

Cystic Fibrosis – Pipeline by ProQR Therapeutics NV, H2 2019

Cystic Fibrosis – Pipeline by Protalix BioTherapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Proteostasis Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Pulmatrix Inc, H2 2019

Cystic Fibrosis – Pipeline by Rare Partners Srl, H2 2019

Cystic Fibrosis – Pipeline by Recode Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Renovion Inc, H2 2019

Cystic Fibrosis – Pipeline by ReveraGen BioPharma Inc, H2 2019

Cystic Fibrosis – Pipeline by Riptide Bioscience Inc, H2 2019

Cystic Fibrosis – Pipeline by Santhera Pharmaceuticals Holding AG, H2 2019

Cystic Fibrosis – Pipeline by SciBac Inc, H2 2019

Cystic Fibrosis – Pipeline by Sequoia Sciences Inc, H2 2019

Cystic Fibrosis – Pipeline by Silurian Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by SolAeroMed Inc, H2 2019

Cystic Fibrosis – Pipeline by Spirovant Sciences Inc, H2 2019

Cystic Fibrosis – Pipeline by Spyryx Biosciences Inc, H2 2019

Cystic Fibrosis – Pipeline by Synedgen Inc, H2 2019

Cystic Fibrosis – Pipeline by Synovo GmbH, H2 2019

Cystic Fibrosis – Pipeline by Synspira LLC, H2 2019

Cystic Fibrosis – Pipeline by Takeda Pharmaceutical Co Ltd, H2 2019

Cystic Fibrosis – Pipeline by TGV-Inhalonix Inc, H2 2019

Cystic Fibrosis – Pipeline by Translate Bio Inc, H2 2019

Cystic Fibrosis – Pipeline by Trucode Gene Repair Inc, H2 2019

Cystic Fibrosis – Pipeline by Vast Therapeutics Inc, H2 2019

Cystic Fibrosis – Pipeline by Vectura Group Plc, H2 2019

Cystic Fibrosis – Pipeline by Verona Pharma Plc, H2 2019

Cystic Fibrosis – Pipeline by Vertex Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Pipeline by Vironika LLC, H2 2019

Cystic Fibrosis – Pipeline by Zambon Co SpA, H2 2019

Cystic Fibrosis – Pipeline by Zata Pharmaceuticals Inc, H2 2019

Cystic Fibrosis – Dormant Projects, H2 2019

Cystic Fibrosis – Discontinued Products, H2 2019

Cystic Fibrosis – Discontinued Products, H2 2019 (Contd..1), H2 2019”

List of Figures

“List of Figures

Number of Products under Development for Cystic Fibrosis, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Number of Products by Top 10 Targets, H2 2019

Number of Products by Stage and Top 10 Targets, H2 2019

Number of Products by Top 10 Mechanism of Actions, H2 2019

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019

Number of Products by Top 10 Routes of Administration, H2 2019

Number of Products by Stage and Top 10 Routes of Administration, H2 2019

Number of Products by Top 10 Molecule Types, H2 2019

Number of Products by Stage and Top 10 Molecule Types, H2 2019”

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