Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | Octapharma, Dicerna, Alnylam, Kamada

Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | Octapharma, Dicerna, Alnylam, Kamada
Delveinsight Business Research LLP
As per DelveInsight’s assessment, globally, about 12+ key pharma and biotech companies are working on 12+ pipeline drugs in the Alpha-1 Antitrypsin Deficiency (AATD) therapeutics landscape based on different Routes of Administration (ROA), Mechanism of Action (MOA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Insight, 2023” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Alpha-1 Antitrypsin Deficiency Market. 

The Alpha-1 Antitrypsin Deficiency Pipeline report embraces in-depth commercial, regulatory, and Alpha-1 Antitrypsin Deficiency clinical trial assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the emerging Alpha-1 Antitrypsin Deficiency drugs, including the mechanism of action, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.

Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Analysis

The report provides insights into: 

  • The report provides detailed insights into the emerging therapies for Alpha-1 Antitrypsin Deficiency treatment and the aggregate therapies developed by major pharma companies.

  • It accesses the different Alpha-1 Antitrypsin Deficiency therapies segmented into early-stage, mid-stage, and late-stage of clinical development.

  • It outlines the major Alpha-1 Antitrypsin Deficiency companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the Alpha-1 Antitrypsin Deficiency drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Alpha-1 Antitrypsin Deficiency therapeutic market.

Alpha-1 Antitrypsin Deficiency (AATD) Therapeutics Landscape

Several major pharma and biotech companies are developing therapies for Alpha-1 Antitrypsin Deficiency. Currently, Deerland Probiotics & Enzymes is leading the therapeutics market with its Alpha-1 Antitrypsin Deficiency drug candidates in the most advanced stage of clinical development.

Alpha-1 Antitrypsin Deficiency (AATD) Companies Actively Working in the Therapeutic Market Include:

  • Octapharma

  • Dicerna Pharmaceuticals

  • Alnylam Pharmaceuticals

  • Arrowhead Pharmaceuticals

  • Vertex Pharmaceuticals

  • AstraZeneca

  • Kamada, Ltd

And Many Others

Emerging and Marketed Alpha-1 Antitrypsin Deficiency (AATD)  Drugs Covered in the Report Include:

  • 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 granted Fast Track designation to ARO-AAT. ARO-AAT is in Phase 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 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.

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Analysis of Emerging Alpha-1 Antitrypsin Deficiency Therapies by Phases

The report covers the emerging products under different phases of clinical development like –

  • Late-stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage product (Phase I) 

  • Pre-clinical and Discovery stage candidates

  • Discontinued and inactive candidates

Route of Administration

Alpha-1 Antitrypsin Deficiency pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Intra-articular

  • Intraocular

  • Intrathecal

  • Intravenous

  • Ophthalmic

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical

  • Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

  • Oligonucleotide

  • Peptide

  • Small molecule

Learn How the Alpha-1 Antitrypsin Deficiency Treatment Outlook will Evolve with the Ongoing Clinical and commercial Activities in the Therapeutic Market @

Table of Content (TOC)

1. Report Introduction

2. Executive Summary

3. Alpha-1 Antitrypsin Deficiency Treatment Patterns

4. Alpha-1 Antitrypsin Deficiency – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Alpha-1 Antitrypsin Deficiency Late Stage Products (Phase-III)

7. Alpha-1 Antitrypsin Deficiency Mid-Stage Products (Phase-II)

8. Alpha-1 Antitrypsin Deficiency Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Alpha-1 Antitrypsin Deficiency Discontinued Products

13. Alpha-1 Antitrypsin Deficiency Product Profiles

14. Major Alpha-1 Antitrypsin Deficiency Companies in the Market

15. Key Products in the Alpha-1 Antitrypsin Deficiency Therapeutics Segment

16. Dormant and Discontinued Products

17. Alpha-1 Antitrypsin Deficiency Unmet Needs

18. Alpha-1 Antitrypsin Deficiency Future Perspectives

19. Alpha-1 Antitrypsin Deficiency Analyst Review  

20. Appendix

21. Report Methodology

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. 

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