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

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    Report

  • 124 Pages
  • April 2022
  • Region: Global
  • Global Markets Direct
  • ID: 5589947
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The 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


The 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under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 1, 7, 2, 24, 5 and 2 respectively.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 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.

Table of Contents

IntroductionReport CoverageAlpha-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 DevelopmentAlpha-1 Antitrypsin Deficiency (A1AD) - Drug ProfilesAlpha-1 Antitrypsin Deficiency (A1AD) - Dormant ProjectsAlpha-1 Antitrypsin Deficiency (A1AD) - Discontinued ProductsAlpha-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
AppendixMethodologyCoverageSecondary ResearchPrimary ResearchExpert Panel ValidationContact the Analyst
List of Tables
  • Number of Products under Development for Alpha-1 Antitrypsin Deficiency (A1AD), 2022
  • Number of Products under Development by Companies, 2022
  • Products under Development by Companies, 2022
  • 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
  • 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

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • ADARx Pharmaceuticals Inc
  • Alnylam Pharmaceuticals Inc
  • Apic Bio Inc
  • Applied Genetic Technologies Corp
  • Arrowhead Pharmaceuticals Inc
  • Beam Therapeutics Inc
  • BioMarin Pharmaceutical Inc
  • Caravella Biopharma SA
  • Centessa Pharmaceuticals Plc
  • Editas Medicine Inc
  • Evolve Biologics Inc
  • Graphite Bio Inc
  • Grifols SA
  • Hepatx Corp
  • Inhibrx Inc
  • Intellia Therapeutics Inc
  • International Stem Cell Corp
  • Kamada Pharmaceuticals
  • Korro Bio Inc
  • Krystal Biotech Inc
  • LEXEO Therapeutics LLC
  • Liminal BioSciences Inc
  • Logicbio Therapeutics Inc
  • Mereo Biopharma Group Plc
  • Moderna Inc
  • Novo Nordisk AS
  • pH Pharma Co Ltd
  • PlantForm Corp
  • Promethera Biosciences SA
  • Renovion Inc
  • Santhera Pharmaceuticals Holding AG
  • Shape Therapeutics Inc
  • Takeda Pharmaceutical Co Ltd
  • Vera Therapeutics Inc
  • Vertex Pharmaceuticals Inc
  • Wave Life Sciences Ltd