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Alpha1-Antitrypsin Deficiency (AATD)

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    Drug Pipelines

  • May 2021
  • Region: Global
  • DelveInsight
  • ID: 5145403
This “Alpha1-antitrypsin deficiency (AATD) - Pipeline Insight, 2021,” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Alpha1-antitrypsin deficiency (AATD) pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

  • Global coverage

Alpha1-antitrypsin deficiency (AATD) Understanding


Alpha1-antitrypsin deficiency (AATD): Overview


Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease. The earliest symptoms of AATD are shortness of breath following mild activity, reduced ability to exercise, and wheezing. Other signs and symptoms can include unintentional weight loss, recurring respiratory infections, fatigue, and rapid heartbeat upon standing. Mutations in the SERPINA1 gene causes alpha-1 antitrypsin deficiency and it is inherited in a codominant manner. Standard medical therapies and supportive care for the specific medical problem are the treatments for AATD. However, augmentation therapy is available to some people with AATD who have lung problems. Alpha1-Proteinase Inhibitor (Prolastin) is a FDA approved treatment for AATD condition.

"Alpha1-antitrypsin deficiency (AATD) - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha1-antitrypsin deficiency (AATD) pipeline landscape is provided which includes the disease overview and Alpha1-antitrypsin deficiency (AATD) treatment guidelines. The assessment part of the report embraces, in depth Alpha1-antitrypsin deficiency (AATD) commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Alpha1-antitrypsin deficiency (AATD) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights


The companies and academics are working to assess challenges and seek opportunities that could influence Alpha1-antitrypsin deficiency (AATD) R&D. The therapies under development are focused on novel approaches to treat/improve Alpha1-antitrypsin deficiency (AATD).

Alpha1-antitrypsin deficiency (AATD) Emerging Drugs Chapters


This segment of the Alpha1-antitrypsin deficiency (AATD) report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Alpha1-antitrypsin deficiency (AATD) Emerging Drugs


ARO AAT: Arrowhead Pharmaceuticals

ARO AAT is a second generation, N-Acetylgalactosamine (GalNAc) based, subcutaneously administered RNA interference based therapeutic. ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. In June 2019, the US FDA has granted Fast Track designation to ARO-AAT. ARO-AAT is in Phase II/III clinical studies for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD).

ALN-AAT02: Alnylam Pharmaceuticals

ALN AAT02 is a subcutaneously administered RNAi therapeutic that targets alpha-1 antitrypsin (AAT). ALN-AAT02 is based on the Alnylam's enhanced stabilization chemistry plus (ESC+)-GalNAc-conjugate technology, which enables subsequent subcutaneous dosing with increased selectivity and a wide therapeutic index. The drug is currently in Phase I/II clinical development for the treatment of ZZ Type AAT Deficiency Liver Disease.

Alpha1-antitrypsin deficiency (AATD): Therapeutic Assessment


This segment of the report provides insights about the different Alpha1-antitrypsin deficiency (AATD) drugs segregated based on following parameters that define the scope of the report, such as:


Major Players in Alpha1-antitrypsin deficiency (AATD)


There are approx. 12+ key companies which are developing the therapies for Alpha1-antitrypsin deficiency (AATD). The companies which have their Alpha1-antitrypsin deficiency (AATD) drug candidates in the most advanced stage, i.e. Phase II/III include, Arrowhead Pharmaceuticals.

Phases


This report covers around 12+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

Alpha1-antitrypsin deficiency (AATD) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Oral
  • Parenteral
  • Intravitreal
  • Subretinal
  • Topical
  • Molecule Type

Products have been categorized under various Molecule types such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Alpha1-antitrypsin deficiency (AATD): Pipeline Development Activities


The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Alpha1-antitrypsin deficiency (AATD) therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities


The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Alpha1-antitrypsin deficiency (AATD) drugs.

Alpha1-antitrypsin deficiency (AATD) Report Insights

  • Alpha1-antitrypsin deficiency (AATD) Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Alpha1-antitrypsin deficiency (AATD) Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Questions Answered


Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Alpha1-antitrypsin deficiency (AATD) drugs?
  • How many Alpha1-antitrypsin deficiency (AATD) drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alpha1-antitrypsin deficiency (AATD)?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Alpha1-antitrypsin deficiency (AATD) therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Alpha1-antitrypsin deficiency (AATD) and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • Alnylam Pharmaceuticals
  • Arrowhead Pharmaceuticals
  • Kamada
  • Mereo BioPharma
  • Santhera Pharmaceuticals
  • Centessa Pharmaceuticals
  • Inhibrx
  • WaVe Life Sciences
  • Promethera Biosciences
  • Beam Therapeutics

Key Products

  • ALN-AAT02
  • ARO AAT
  • Recombinant alpha-1 antitrypsin
  • Alvelestat
  • Lonodelestat
  • ZF874
  • INBRX 101
  • SERPINA1 stereopure nucleic acid therapeutic

Research programme: stem cell therapies

Research programme: DNA base editor therapeutics

Table of Contents

Introduction

Executive Summary

Alpha1-antitrypsin deficiency (AATD): Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics
  • Comparative Analysis

Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Alpha1-antitrypsin deficiency (AATD) - Analytical Perspective

In-depth Commercial Assessment
  • Alpha1-antitrypsin deficiency (AATD) companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Alpha1-antitrypsin deficiency (AATD) Collaboration Deals
  • Company-Company Collaborations (Licensing/Partnering) Analysis
  • Company-University Collaborations (Licensing/Partnering) Analysis

Late Stage Products (Phase III)
  • Comparative Analysis

Drug name: Company name
  • Product Description
  • Research and Development
  • Product Development Activities

Mid Stage Products (Phase II/III)
  • Comparative Analysis

ARO AAT: Arrowhead Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities

Early Stage Products (Phase I/II)
  • Comparative Analysis

ALN-AAT02: Alnylam Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities

Preclinical/Discovery Stage Products
  • Comparative Analysis

Recombinant alpha-1 antitrypsin: Kamada
  • Product Description
  • Research and Development
  • Product Development Activities

Inactive Products
  • Comparative Analysis

Alpha1-antitrypsin deficiency (AATD) Key Companies

Alpha1-antitrypsin deficiency (AATD) Key Products

Alpha1-antitrypsin deficiency (AATD)- Unmet Needs

Alpha1-antitrypsin deficiency (AATD)- Market Drivers and Barriers

Alpha1-antitrypsin deficiency (AATD)- Future Perspectives and Conclusion

Alpha1-antitrypsin deficiency (AATD) Analyst Views

Alpha1-antitrypsin deficiency (AATD) Key Companies

AppendixList of Tables
Table 1 Total Products for Alpha1-antitrypsin deficiency (AATD)
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Alpha1-antitrypsin deficiency (AATD)
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Alnylam Pharmaceuticals
  • Arrowhead Pharmaceuticals
  • Kamada
  • Mereo BioPharma
  • Santhera Pharmaceuticals
  • Centessa Pharmaceuticals
  • Inhibrx
  • WaVe Life Sciences
  • Promethera Biosciences
  • Beam Therapeutics