嚢胞性線維症：パイプライン製品の分析

嚢胞性線維症は、粘液および発汗、消化液を生成する細胞に作用する遺伝性疾患です。その症状は喘鳴、息切れ、度重なる肺感染症、鼻腔の炎症・鼻詰まり、重度の便秘などがあります。嚢胞性線維症に罹患には遺伝的要因、人種（北欧圏の白人に多い）が深くかかわっていると言われています。治療には抗生物質、濃性粘液の分解薬、気管支拡張薬、経口の膵酵素剤などを利用します。

当レポートでは、嚢胞性線維症治療薬の開発状況について調査しており、パイプライン製品の概要、治験の段階別の製品概要、主要企業のプロファイル、薬剤のプロファイル、パイプライン製品の最新動向などを提供しています。

イントロダクション

• 調査範囲

嚢胞性線維症の概要

治療薬の開発

嚢胞性線維症：治療薬の評価

嚢胞性線維症の治療薬開発に従事している企業

薬剤プロファイル

嚢胞性線維症：休止中のプロジェクト

嚢胞性線維症：開発が中止された製品

嚢胞性線維症：製品開発のマイルストーン

付録

図表

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

• 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

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Cystic Fibrosis - Pipeline Review, H2 2019, provides an overview of the Cystic Fibrosis (Respiratory) pipeline landscape.

Cystic fibrosis (CF) is an inherited disease that causes mucus to build up and clog some of the body's organs, particularly the lungs and pancreas. Cystic fibrosis affects the cells that produce mucus, sweat and digestive juices. Symptoms include wheezing, breathlessness, repeated lung infections, inflamed nasal passages or a stuffy nose and severe constipation. Risk factors include family history and race (Northern European ancestry). Treatment includes antibiotics, mucus-thinning, bronchodilators and oral pancreatic enzymes.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Cystic Fibrosis - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Cystic Fibrosis (Respiratory), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Cystic Fibrosis (Respiratory) pipeline guide also reviews of key players involved in therapeutic development for Cystic Fibrosis and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 1, 5, 34, 18, 66 and 33 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical, Discovery and Unknown stages comprises 2, 1, 10, 9 and 1 molecules, respectively.

Cystic Fibrosis (Respiratory) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope:

• The pipeline guide provides a snapshot of the global therapeutic landscape of Cystic Fibrosis (Respiratory).
• The pipeline guide reviews pipeline therapeutics for Cystic Fibrosis (Respiratory) by companies and universities/research institutes based on information derived from company and industry-specific sources.
• The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
• The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
• The pipeline guide reviews key companies involved in Cystic Fibrosis (Respiratory) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Cystic Fibrosis (Respiratory) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
• The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
• The pipeline guide reviews latest news related to pipeline therapeutics for Cystic Fibrosis (Respiratory)

Reasons to buy:

• Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
• Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
• Find and recognize significant and varied types of therapeutics under development for Cystic Fibrosis (Respiratory).
• Classify potential new clients or partners in the target demographic.
• Develop tactical initiatives by understanding the focus areas of leading companies.
• Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
• Formulate corrective measures for pipeline projects by understanding Cystic Fibrosis (Respiratory) 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.
• Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

• Introduction
• Cystic Fibrosis - Overview
• Cystic Fibrosis - Therapeutics Development
• Cystic Fibrosis - Therapeutics Assessment
• Cystic Fibrosis - Companies Involved in Therapeutics Development
• Cystic Fibrosis - Drug Profiles
• Cystic Fibrosis - Dormant Projects
• Cystic Fibrosis - Discontinued Products
• Cystic Fibrosis - Product Development Milestones
• Appendix