Alpha 1 Antitrypsin Deficiency Liver Disease Pipeline Insight

DelveInsight’s, “Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease – Pipeline Insights, 2021,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Understanding

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Overview

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is a common genetic cause of liver disease, with the most severe mutation found in 1:3500 live births. A1AT is a large, 52‐kDa serum glycoprotein abundantly produced by the liver. The deficiency is caused by mutation in the A1AT protein encoding gene SERPINA1 and is inherited in an autosomal recessive manner. Mutations in SERPINA1 leads to the formation of harmful aggregates of mutant A1AT protein in the ER of hepatocytes leading to inflammation, fibrosis, cirrhosis, and increased risk of hepatocellular carcinoma in adults. Risk factors for progression of A1ATD liver disease included male sex, age older than 50 years, viral hepatitis, and diabetes.

Symptoms

The symptoms of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease depends on the condition and the age at which they appear vary among individuals. The earliest symptoms are shortness of breath following mild activity, reduced ability to exercise, and wheezing, unintentional weight loss, recurring respiratory infections, fatigue, and rapid heartbeat upon standing. Affected individuals often develop emphysema, which is a lung disease caused by damage to the small air sacs in the lungs (alveoli). Symptoms of A1ATD include jaundice, dark urine, itchy skin and bleeding. The liver is gradually scarred and liver function can decline over time. About 10 percent of infants with alpha-1 antitrypsin deficiency develop liver disease, which often causes yellowing of the skin and whites of the eyes (jaundice). In rare cases, people with alpha-1 antitrypsin deficiency develop a skin condition called panniculitis, which is characterized by hardened skin with painful lumps or patches.

Diagnosis

Diagnosis of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is underreported and underdiagnosed, often with a diagnostic delay of 5 to 10 years.


The diagnosis of A1ATD may require biopsies, blood tests, CAT scans and ultrasound. Following diagnosis, patients found to have A1ATD are largely managed supportively with close monitoring for progression of liver disease like fibrosis or HCC. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Anti-human A1AT antibody can also confirm the presence of ATZ globules. Electron microscopy studies can identify multiple globular inclusions within dilated, congested rough ER cisterns, adjacent to secondary lysosomes.

Treatment

Treatment options for the A1ATD lung disease are based on the severity of the liver affected and the stage of diagnosis. The treatment regimen may include Chaperones Autophagy-Enhancing agents, Bile acids as small molecule therapy, agents inhibiting ATZ polymerization, Gene therapy and editing, and Cell therapy. Sometimes a liver transplant may be needed if significant liver damage has been caused. It is important to avoid all contact with smoking because lung disease (emphysema) develops over the second decade of life.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Emerging Drugs Chapters

This segment of the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Emerging Drugs

  • DCR-A1AT: Dicerna Pharmaceuticals

DCR-A1AT is a ribonucleic acid interference (RNAi) therapeutic, investigated for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency. DCR-A1AT utilizes Dicerna’s GalXCTM technology, which enables subcutaneous delivery and optimizes the activity of the RNAi pathway so that it operates in the most specific and potent fashion.


  • APB-101: Apic Bio

APB-101 is a “liver-sparing” gene therapy designed as a one-time treatment for Alpha-1 patients. APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. In pre-clinical studies the molecule has demonstrated the ability to reduce levels of the mutant Alpha-1 protein (Z-AAT) and at the same time program liver cells to produce the correct Alpha-1 protein (M-AAT).

Further product details are provided in the report……..

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Therapeutic Assessment

This segment of the report provides insights about the different Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs segregated based on following parameters that define the scope of the report, such as:


  • Major Players in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease

There are approx. 5+ key companies which are developing the therapies for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease. The companies which have their Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drug candidates in the most advanced stage, i.e. phase II/III include, Arrowhead Pharmaceuticals.


  • Phases

DelveInsight’s report covers around 5+ 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


Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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

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


Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs.

Report Highlights

  • The companies and academics are working to assess challenges and seek opportunities that could influence Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease R&D. The therapies under development are focused on novel approaches to treat/improve Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease.
  • On 6th April 2020, Alnylam Pharmaceuticals and Dicerna Pharmaceuticals announced the development and commercialization collaboration on investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease (alpha-1 liver disease).


Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Report Insights

  • Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs


Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Report Assessment

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

Key Questions

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs?
  • How many Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • Alnylam Pharmaceuticals
  • Dicerna Pharmaceuticals
  • Novartis
  • Apic Bio
  • Vertex Pharmaceuticals
  • Arrowhead Pharmaceuticals

Key Products

  • ALN-AAT02
  • DCR-A1AT
  • Carbamazepine
  • APB-101
  • VX-864
  • TAK-999

 

Introduction

Executive Summary

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: 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

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

• Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Collaboration Deals

• Company-Company Collaborations (Licensing / Partnering) Analysis

• Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Phase II/II)

• Comparative Analysis

TAK-999: Arrowhead Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Mid Stage Products (Phase II)

• Comparative Analysis

VX-864: Vertex Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II)

• Comparative Analysis

DCR-A1AT: Dicerna Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Pre-clinical and Discovery Stage Products

• Comparative Analysis

APB-101: Apic Bio

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

• Comparative Analysis

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Key Companies

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Key Products

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Unmet Needs

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Market Drivers and Barriers

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Future Perspectives and Conclusion

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Analyst Views

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Key Companies

Appendix

List of Table

 Table 1 Total Products for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease

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 Products

List of Figures

 Figure 1 Total Products for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease

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

• Alnylam Pharmaceuticals

• Dicerna Pharmaceuticals

• Novartis

• Apic Bio

• Vertex Pharmaceuticals

• Arrowhead Pharmaceuticals

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