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Alpha-1 Antitrypsin Deficiency Pipeline 2025: FDA Updates, Therapy Innovations, and Clinical Trial Landscape Analysis by DelveInsight | Apic Bio, Dicerna Pharma, Peak Bio, Arrowhead Pharma

04-10-2025 10:18 PM CET | Health & Medicine

Press release from: DelveInisight Business Research

Alpha-1 Antitrypsin Deficiency Pipeline 2025: FDA Updates,

Alpha-1 Antitrypsin Deficiency pipeline constitutes 12+ key companies continuously working towards developing 12+ Alpha-1 Antitrypsin Deficiency treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Alpha-1 Antitrypsin Deficiency Pipeline Insight, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Alpha-1 Antitrypsin Deficiency Market.

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

Some of the key takeaways from the Alpha-1 Antitrypsin Deficiency Pipeline Report: https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Alpha-1 Antitrypsin Deficiency treatment therapies with a considerable amount of success over the years. Alpha-1 Antitrypsin Deficiency Key players such as - Apic Bio, Dicerna Pharmaceuticals, Peak Bio, Arrowhead Pharmaceuticals, and others, are developing therapies for the Alpha-1 Antitrypsin Deficiency treatment
• Alpha-1 Antitrypsin Deficiency Emerging therapies such as - APB-101, Belcesiran, PHP 303, ARO-AAT, and others are expected to have a significant impact on the Alpha-1 Antitrypsin Deficiency market in the coming years.
• In March 2025, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines via base editing, has reported initial safety and efficacy data from its Phase 1/2 trial of BEAM-302. The findings establish a clinical proof-of-concept for BEAM-302 as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and in vivo base editing. Preliminary results from the first three single-ascending dose cohorts indicate that BEAM-302 was well tolerated, with single doses demonstrating a durable, dose-dependent correction of the disease-causing mutation.
• In November 2024, Korro Bio has received approval in Australia to begin the two-part Phase I/IIa REWRITE trial for its investigational treatment, KRRO-110, aimed at addressing alpha-1 antitrypsin deficiency (AATD). The approvals were granted by the Australian Bellberry Human Research Ethics Committee (HREC) and the Therapeutic Goods Administration (TGA).
• In October 2024, Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on RNA-based therapies, has announced positive proof-of-mechanism data from its ongoing Phase 1b/2a RestorAATion-2 study of WVE-006 for alpha-1 antitrypsin deficiency (AATD). WVE-006 is a GalNAc-conjugated, subcutaneously administered A-to-I RNA editing oligonucleotide (AIMer), developed using Wave's advanced oligonucleotide chemistry platform. It is specifically designed to target lung and liver diseases associated with AATD.
• In June 2024, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company focused on developing precision genetic medicines through base editing, has announced the treatment of the first patient with BEAM-302. This investigational in vivo base editing therapy is designed to precisely correct the root cause of severe alpha-1 antitrypsin deficiency (AATD) and is currently being assessed in a Phase 1/2 clinical trial.
• In April 2024, Enlivex Therapeutics announced promising topline safety and efficacy results from its Phase II clinical trial of Allocetra, an off-the-shelf cell therapy for sepsis. The randomized, placebo-controlled trial aims to identify the optimal dose of Allocetra when administered in conjunction with the standard care for sepsis caused by different infections.

Alpha-1 Antitrypsin Deficiency Overview
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by a deficiency or malfunction of alpha-1 antitrypsin (AAT) protein, which is produced primarily in the liver. AAT is important for protecting the lungs from damage caused by enzymes produced by white blood cells, specifically neutrophil elastase.

Get a Free sample for the Alpha-1 Antitrypsin Deficiency Market Forecast, Size & Share Analysis Report:
https://www.delveinsight.com/report-store/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

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

Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics Assessment
• Alpha-1 Antitrypsin Deficiency Assessment by Product Type
• Alpha-1 Antitrypsin Deficiency By Stage and Product Type
• Alpha-1 Antitrypsin Deficiency Assessment by Route of Administration
• Alpha-1 Antitrypsin Deficiency By Stage and Route of Administration
• Alpha-1 Antitrypsin Deficiency Assessment by Molecule Type
• Alpha-1 Antitrypsin Deficiency by Stage and Molecule Type

DelveInsight's Alpha-1 Antitrypsin Deficiency Report covers around 4+ 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 & Inactive candidates
• Route of Administration

Further Alpha-1 Antitrypsin Deficiency product details are provided in the report. Download the Alpha-1 Antitrypsin Deficiency pipeline report to learn more about the emerging Alpha-1 Antitrypsin Deficiency therapies
https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Emerging Alpha-1 Antitrypsin Deficiency Drugs Under Different Phases of Clinical Development Include:
• APB-101: Apic Bio
• Belcesiran: Dicerna Pharmaceuticals
• PHP 303: Peak Bio
• ARO-AAT: Arrowhead Pharmaceuticals

Alpha-1 Antitrypsin Deficiency Pipeline Analysis:
The Alpha-1 Antitrypsin Deficiency pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the Alpha-1 Antitrypsin Deficiency treatment with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Alpha-1 Antitrypsin Deficiency Treatment.
• Alpha-1 Antitrypsin Deficiency key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Alpha-1 Antitrypsin Deficiency Drugs 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.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Alpha-1 Antitrypsin Deficiency market.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Alpha-1 Antitrypsin Deficiency drugs and therapies
https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Alpha-1 Antitrypsin Deficiency Pipeline Market Drivers
• Increasing investment in the R&D activities for developing novel therapeutics
• Rise in awareness among people

Alpha-1 Antitrypsin Deficiency Pipeline Market Barriers
• Lack of diagnostic methods
• Treatment dosage being poorly standardized

Scope of Alpha-1 Antitrypsin Deficiency Pipeline Drug Insight
• Coverage: Global
• Key Alpha-1 Antitrypsin Deficiency Companies: Apic Bio, Dicerna Pharmaceuticals, Peak Bio, Arrowhead Pharmaceuticals, and others
• Key Alpha-1 Antitrypsin Deficiency Therapies: APB-101, Belcesiran, PHP 303, ARO-AAT, and others
• Alpha-1 Antitrypsin Deficiency Therapeutic Assessment: Alpha-1 Antitrypsin Deficiency current marketed and Alpha-1 Antitrypsin Deficiency emerging therapies
• Alpha-1 Antitrypsin Deficiency Market Dynamics: Alpha-1 Antitrypsin Deficiency market drivers and Alpha-1 Antitrypsin Deficiency market barriers

Request for Sample PDF Report for Alpha-1 Antitrypsin Deficiency Pipeline Assessment and clinical trials
https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Table of Contents
1 Alpha-1 Antitrypsin Deficiency Report Introduction
2 Alpha-1 Antitrypsin Deficiency Executive Summary
3 Alpha-1 Antitrypsin Deficiency Overview
4 Alpha-1 Antitrypsin Deficiency- Analytical Perspective In-depth Commercial Assessment
5 Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics
6 Alpha-1 Antitrypsin Deficiency Late Stage Products (Phase II/III)
7 Alpha-1 Antitrypsin Deficiency Mid Stage Products (Phase II)
8 Alpha-1 Antitrypsin Deficiency Early Stage Products (Phase I)
9 Alpha-1 Antitrypsin Deficiency Preclinical Stage Products
10 Alpha-1 Antitrypsin Deficiency Therapeutics Assessment
11 Alpha-1 Antitrypsin Deficiency Inactive Products
12 Company-University Collaborations (Licensing/Partnering) Analysis
13 Alpha-1 Antitrypsin Deficiency Key Companies
14 Alpha-1 Antitrypsin Deficiency Key Products
15 Alpha-1 Antitrypsin Deficiency Unmet Needs
16 Alpha-1 Antitrypsin Deficiency Market Drivers and Barriers
17 Alpha-1 Antitrypsin Deficiency Future Perspectives and Conclusion
18 Alpha-1 Antitrypsin Deficiency Analyst Views
19 Appendix
20 About DelveInsight

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DelveInsight's 'Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast-2034' report delivers an in-depth understanding of the 7MM, historical and forecasted epidemiology as well as the 7MM market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom),

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DelveInsight's 'Alpha-1 Antitrypsin Deficiency Epidemiology Forecast to 2032' report delivers an in-depth understanding of the disease, historical and forecasted Cholangiocarcinoma epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Contact Us:
Gaurav Bora
gbora@delveinsight.com
+14699457679
www.delveinsight.com

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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