Los Angeles, USA, Aug. 12, 2021 (GLOBE NEWSWIRE) -- Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline: Insights into New Pipeline Therapies and Landscape; Future Key Pharma Companies and Novel Therapeutic Assessment
Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline is in its nascent budding stage with novel molecules targeting new therapeutic targets.
DelveInsight’s ‘Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Pipeline Insight report provides comprehensive insights to Alpha-1 Antitrypsin Deficiency Liver Disease pipeline therapies and key companies involved in the production. The available marketed therapies in A1ATD Liver Disease pipeline in different phases of clinical development, comparative analysis and future growth potential of the A1ATD Liver Disease pipeline territory. Overview of A1ATD Liver Disease including its Causes, Symptoms, Risk Factors, Pathophysiology, Diagnosis and Treatment are covered. The report also builds an understanding of the limitations and challenges in the field of Alpha-1 Antitrypsin Deficiency Liver Disease.
Some of the key highlights from the Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline report:
- DelveInsight’s analysis presents Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline with 10+ active players in the domain investigating 10+ pipeline therapies.
- Many key companies such as Apic Bio, Centessa Pharmaceuticals, Dicerna Pharmaceuticals, PH Pharmaceutical, Arrowhead Pharmaceuticals, Alnylam Pharmaceuticals, and others are proactively engaged in the development of the potential drug candidates to improve the ongoing Alpha-1 Antitrypsin Deficiency Liver Disease treatment.
- Emerging Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline therapies such as APB-101, ZF874, Belcesiran, PHP 303, ARO-AAT, ALN-AAT02, and others are under investigation in different phases of clinical trials.
- In July 2021, Arrowhead Pharmaceuticals announced that it was granted Breakthrough Therapy designation (BTD) by the US FDA for ARO-AAT, also known as TAK-999, being co-developed with Takeda Pharmaceutical Company as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
- In July 2021, Dicerna Pharmaceuticals announced interim results from the four completed active-treatment dose cohorts (0.1, 1.0, 3.0 and 6.0 mg/kg) of its Phase I double-blind, placebo-controlled, randomized trial of belcesiran, an investigational GalXC™ RNAi therapeutic in development for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (AATLD).
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The Alpha-1 Antitrypsin Deficiency Liver Disease pipeline report puts forward detailed profiles of the pipeline assets along with the inactive and dormant assets, comparative analysis of clinical and non-clinical stage Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease products, comprehensive assessment of driving and restraining factors along with the opportunities and risks in the Alpha-1 Antitrypsin Deficiency Liver Disease pipeline landscape.
Alpha-1 Antitrypsin Deficiency Liver Disease Overview
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease, is an inherited metabolic disorder in which this protein is absent or defective. The deficiency is a result of a mutation in the A1AT protein encoding gene SERPINA1 and is inherited in an autosomal recessive manner. Alpha-1 antitrypsin is a large, 52‐kDa serum glycoprotein and a serine protease inhibitor.
Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline Drugs
| Drug | Company | Phase | MoA | RoA |
| APB-101 | Apic Bio | Preclinical | Gene transference | NA |
| ZF874 | Centessa Pharmaceuticals | Phase I | Alpha 1 antitrypsin modulators | Oral |
| Belcesiran | Dicerna Pharmaceuticals | Phase II | Alpha 1 antitrypsin modulator | Subcutaneous |
| PHP 303 | PH Pharmaceuticals | Phase II | Neutrophil elastase (NE) inhibitor | Oral |
| ARO-AAT | Arrowhead Pharmaceuticals | Phase II//III | Alpha 1 antitrypsin inhibitors | Subcutaneous |
| ALN-AAT02 | Alnylam Pharmaceuticals | Phase I/II | Alpha 1 antitrypsin modulators | Subcutaneous |
| LB 201 | LogicBio Therapeutics | Discovery | Gene transference | NA |
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Alpha-1 Antitrypsin Deficiency Liver Disease Therapeutics Assessment
The Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline report proffers an integral view of the Alpha-1 Antitrypsin Deficiency Liver Disease emerging novel therapies segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Mechanism of Action.
By Product Type
- Mono
- Combination
By Stage
- Discovery
- Pre-clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
By Route of Administration
- Subcutaneous
- Intravenous
- Oral
- Inhalation
By Molecule Type
- Gene therapy
- Stem cell therapy
- Small molecules
- RNAi Molecule
By Mechanism of Action
- Gene transference
- Alpha 1 antitrypsin modulators
- Neutrophil elastase (NE) inhibitor
- Sodium channel antagonists
By Targets
- Alpha 1 antitrypsin
- Neutrophil elastase
- Sodium channel
Scope of the Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline Report
- Coverage: Global
- Major Players: Apic Bio, Centessa Pharmaceuticals, Dicerna Pharmaceuticals, PH Pharmaceutical, Arrowhead Pharmaceuticals, LogicBio Therapeutics, Alnylam Pharmaceuticals.
- Key Alpha-1 Antitrypsin Deficiency Liver Disease Pipeline Theraṣpies: APB-101, ZF874, Belcesiran, PHP 303, ARO-AAT, LB 201, ALN-AAT02.
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Table of Contents
| 1 | Introduction |
| 2 | Executive Summary |
| 3 | Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Overview |
| 4 | Pipeline Therapeutics |
| 5 | Late Stage Products (Pre-Registration) |
| 6 | Late Stage Products (Phase III) |
| 7 | Mid Stage Products (Phase II/III) ARO-AAT: Arrowhead Pharmaceuticals |
| 8 | Early Stage Products (Phase I) ZF874: Centessa Pharmaceuticals |
| 9 | Pre-clinical and Discovery Stage Products APB-101: Apic Bio |
| 10 | Therapeutic Assessment |
| 11 | Inactive Products |
| 12 | Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Unmet Needs |
| 13 | Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Market Drivers and Barriers |
| 14 | Appendix |
| 15 | About DelveInsight |
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