Alpha-1 Antitrypsin Deficiency (A1AD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

April 2022 | 124 pages | ID: A4DAEFF45D79EN
Global Markets Direct

Abstracts

Alpha-1 Antitrypsin Deficiency (A1AD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Alpha-1 Antitrypsin Deficiency (A1AD) - Drugs In Development, 2022, provides an overview of the Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) pipeline landscape.

Alpha-antitrypsin deficiency is an inherited disease that occurs due to lack of alpha-1 antitrypsin (AAT), a protein that protects the lungs. Symptoms include shortness of breath and wheezing, lung infections, rapid heartbeat, weight loss and vision problems. Predisposing factors include smoking, exposure to dust, chemical fumes and infections.

REPORT HIGHLIGHTS

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Alpha-1 Antitrypsin Deficiency (A1AD) - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders), 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 Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Alpha-1 Antitrypsin Deficiency (A1AD) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 1, 7, 2, 24, 5 and 2 respectively.

Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) 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 Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders).
• The pipeline guide reviews pipeline therapeutics for Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) 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 Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) 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 Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders)

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 Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders).
• 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 Alpha-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) 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.

Contents

Introduction
Global Markets Direct Report Coverage
Alpha-1 Antitrypsin Deficiency (A1AD) - Overview
Alpha-1 Antitrypsin Deficiency (A1AD) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Products under Development by Companies
Alpha-1 Antitrypsin Deficiency (A1AD) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Alpha-1 Antitrypsin Deficiency (A1AD) - Companies Involved in Therapeutics Development
Alpha-1 Antitrypsin Deficiency (A1AD) - Drug Profiles
Alpha-1 Antitrypsin Deficiency (A1AD) - Dormant Projects
Alpha-1 Antitrypsin Deficiency (A1AD) - Discontinued Products
Alpha-1 Antitrypsin Deficiency (A1AD) - Product Development Milestones
Featured News & Press Releases
Apr 05, 2022: Inhibrx to present INBRX-101 data at ATS 2022 Annual Meeting
Mar 04, 2022: US FDA grants orphan drug status to Inhibrx’s AATD treatment
Mar 03, 2022: Inhibrx receives FDA Orphan-Drug Designation for INBRX-101 for the treatment of Alpha-1 Antitrypsin Deficiency
Mar 03, 2022: Mereo BioPharma to hold virtual R&D day on monday, march 14, 2022
Nov 12, 2021: Arrowhead presents additional clinical data on investigational ARO-AAT Treatment at AASLD Liver Meeting
Nov 12, 2021: Dicerna Presents data from phase 1 trial of belcesiran at American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2021
Nov 01, 2021: Centessa Pharmaceuticals demonstrates proof-of-mechanism from first three PiMZ subjects dosed in part B of phase 1 study evaluating ZF874
Oct 26, 2021: Mereo BioPharma receives U.S. Orphan Drug Designation for alvelestat in the treatment of alpha-1 antitrypsin deficiency
Oct 14, 2021: Krystal Biotech announces virtual presentation of pre-clinical data on KB408 for the treatment of alpha-1 antitrypsin deficiency at ESGCT
Oct 14, 2021: Dicerna Announces Poster Presentation on Belcesiran at American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® 2021 in November
Oct 12, 2021: INBRX-101 shows favorable safety profile in patients with alpha-1 antitrypsin deficiency and demonstrates the potential to achieve normal alpha-1 antitrypsin levels with monthly dosing
Oct 11, 2021: Inhibrx to host webcast presentation of interim results from the phase 1 trial of INBRX-101, its recombinant human AAT-Fc protein for the treatment of alpha-1 antitrypsin deficiency
Sep 28, 2021: Wave Life Sciences announces new data for leading RNA editing capability across multiple tissues and provides update on AATD program during analyst and investor research webcast
Jul 29, 2021: Arrowhead Pharmaceuticals receives Breakthrough Therapy designation from U.S. FDA for ARO-AAT for the treatment of alpha-1 antitrypsin deficiency associated liver disease
Jul 21, 2021: Dicerna announces interim results from phase 1 trial of Belcesiran for treatment of alpha-1 antitrypsin deficiency-associated liver disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development for Alpha-1 Antitrypsin Deficiency (A1AD), 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022 (Contd..2)
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by ADARx Pharmaceuticals Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Alnylam Pharmaceuticals Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Apic Bio Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Applied Genetic Technologies Corp, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Arrowhead Pharmaceuticals Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Beam Therapeutics Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by BioMarin Pharmaceutical Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Caravella Biopharma SA, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Centessa Pharmaceuticals Plc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Editas Medicine Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Evolve Biologics Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Graphite Bio Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Grifols SA, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Hepatx Corp, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Inhibrx Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Intellia Therapeutics Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by International Stem Cell Corp, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Kamada Pharmaceuticals, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Korro Bio Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Krystal Biotech Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by LEXEO Therapeutics LLC, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Liminal BioSciences Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Logicbio Therapeutics Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Mereo Biopharma Group Plc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Moderna Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Novo Nordisk AS, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by pH Pharma Co Ltd, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by PlantForm Corp, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Promethera Biosciences SA, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Renovion Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Santhera Pharmaceuticals Holding AG, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Shape Therapeutics Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Takeda Pharmaceutical Co Ltd, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Vera Therapeutics Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Vertex Pharmaceuticals Inc, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline by Wave Life Sciences Ltd, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Dormant Projects, 2022
Alpha-1 Antitrypsin Deficiency (A1AD) - Discontinued Products, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development for Alpha-1 Antitrypsin Deficiency (A1AD), 2022
Number of Products under Development by Companies, 2022
Number of Products by Targets, 2022
Number of Products by Stage and Targets, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Top 10 Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022