DelveInsight’s, “Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease – Pipeline Insight, 2020,” 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