This “Alpha1-antitrypsin deficiency (AATD) - Pipeline Insight, 2021,” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Alpha1-antitrypsin deficiency (AATD) 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.
Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease. The earliest symptoms of AATD are shortness of breath following mild activity, reduced ability to exercise, and wheezing. Other signs and symptoms can include unintentional weight loss, recurring respiratory infections, fatigue, and rapid heartbeat upon standing. Mutations in the SERPINA1 gene causes alpha-1 antitrypsin deficiency and it is inherited in a codominant manner. Standard medical therapies and supportive care for the specific medical problem are the treatments for AATD. However, augmentation therapy is available to some people with AATD who have lung problems. Alpha1-Proteinase Inhibitor (Prolastin) is a FDA approved treatment for AATD condition.
"Alpha1-antitrypsin deficiency (AATD) - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha1-antitrypsin deficiency (AATD) pipeline landscape is provided which includes the disease overview and Alpha1-antitrypsin deficiency (AATD) treatment guidelines. The assessment part of the report embraces, in depth Alpha1-antitrypsin deficiency (AATD) commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Alpha1-antitrypsin deficiency (AATD) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
The companies and academics are working to assess challenges and seek opportunities that could influence Alpha1-antitrypsin deficiency (AATD) R&D. The therapies under development are focused on novel approaches to treat/improve Alpha1-antitrypsin deficiency (AATD).
This segment of the Alpha1-antitrypsin deficiency (AATD) 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.
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 has granted Fast Track designation to ARO-AAT. ARO-AAT is in Phase II/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 the 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.
There are approx. 12+ key companies which are developing the therapies for Alpha1-antitrypsin deficiency (AATD). The companies which have their Alpha1-antitrypsin deficiency (AATD) drug candidates in the most advanced stage, i.e. Phase II/III include, Arrowhead Pharmaceuticals.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Alpha1-antitrypsin deficiency (AATD) therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Alpha1-antitrypsin deficiency (AATD) drugs.
Research programme: stem cell therapies
Research programme: DNA base editor therapeutics
Geography Covered
- Global coverage
Alpha1-antitrypsin deficiency (AATD) Understanding
Alpha1-antitrypsin deficiency (AATD): Overview
Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease. The earliest symptoms of AATD are shortness of breath following mild activity, reduced ability to exercise, and wheezing. Other signs and symptoms can include unintentional weight loss, recurring respiratory infections, fatigue, and rapid heartbeat upon standing. Mutations in the SERPINA1 gene causes alpha-1 antitrypsin deficiency and it is inherited in a codominant manner. Standard medical therapies and supportive care for the specific medical problem are the treatments for AATD. However, augmentation therapy is available to some people with AATD who have lung problems. Alpha1-Proteinase Inhibitor (Prolastin) is a FDA approved treatment for AATD condition.
"Alpha1-antitrypsin deficiency (AATD) - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha1-antitrypsin deficiency (AATD) pipeline landscape is provided which includes the disease overview and Alpha1-antitrypsin deficiency (AATD) treatment guidelines. The assessment part of the report embraces, in depth Alpha1-antitrypsin deficiency (AATD) commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Alpha1-antitrypsin deficiency (AATD) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Alpha1-antitrypsin deficiency (AATD) R&D. The therapies under development are focused on novel approaches to treat/improve Alpha1-antitrypsin deficiency (AATD).
Alpha1-antitrypsin deficiency (AATD) Emerging Drugs Chapters
This segment of the Alpha1-antitrypsin deficiency (AATD) 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.
Alpha1-antitrypsin deficiency (AATD) Emerging Drugs
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 has granted Fast Track designation to ARO-AAT. ARO-AAT is in Phase II/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 the 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.
Alpha1-antitrypsin deficiency (AATD): Therapeutic Assessment
This segment of the report provides insights about the different Alpha1-antitrypsin deficiency (AATD) drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Alpha1-antitrypsin deficiency (AATD)
There are approx. 12+ key companies which are developing the therapies for Alpha1-antitrypsin deficiency (AATD). The companies which have their Alpha1-antitrypsin deficiency (AATD) drug candidates in the most advanced stage, i.e. Phase II/III include, Arrowhead Pharmaceuticals.
Phases
This report covers around 12+ 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
Alpha1-antitrypsin deficiency (AATD) 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
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Alpha1-antitrypsin deficiency (AATD): Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Alpha1-antitrypsin deficiency (AATD) 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 Alpha1-antitrypsin deficiency (AATD) drugs.
Alpha1-antitrypsin deficiency (AATD) Report Insights
- Alpha1-antitrypsin deficiency (AATD) Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Alpha1-antitrypsin deficiency (AATD) Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Alpha1-antitrypsin deficiency (AATD) drugs?
- How many Alpha1-antitrypsin deficiency (AATD) drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alpha1-antitrypsin deficiency (AATD)?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Alpha1-antitrypsin deficiency (AATD) 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 Alpha1-antitrypsin deficiency (AATD) and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Alnylam Pharmaceuticals
- Arrowhead Pharmaceuticals
- Kamada
- Mereo BioPharma
- Santhera Pharmaceuticals
- Centessa Pharmaceuticals
- Inhibrx
- WaVe Life Sciences
- Promethera Biosciences
- Beam Therapeutics
Key Products
- ALN-AAT02
- ARO AAT
- Recombinant alpha-1 antitrypsin
- Alvelestat
- Lonodelestat
- ZF874
- INBRX 101
- SERPINA1 stereopure nucleic acid therapeutic
Research programme: stem cell therapies
Research programme: DNA base editor therapeutics
Table of Contents
IntroductionExecutive Summary
Alpha1-antitrypsin deficiency (AATD): 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
Alpha1-antitrypsin deficiency (AATD) - Analytical Perspective
In-depth Commercial Assessment
- Alpha1-antitrypsin deficiency (AATD) companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Alpha1-antitrypsin deficiency (AATD) Collaboration Deals
- Company-Company Collaborations (Licensing/Partnering) Analysis
- Company-University Collaborations (Licensing/Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II/III)
- Comparative Analysis
ARO AAT: Arrowhead Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I/II)
- Comparative Analysis
ALN-AAT02: Alnylam Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Preclinical/Discovery Stage Products
- Comparative Analysis
Recombinant alpha-1 antitrypsin: Kamada
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Alpha1-antitrypsin deficiency (AATD) Key Companies
Alpha1-antitrypsin deficiency (AATD) Key Products
Alpha1-antitrypsin deficiency (AATD)- Unmet Needs
Alpha1-antitrypsin deficiency (AATD)- Market Drivers and Barriers
Alpha1-antitrypsin deficiency (AATD)- Future Perspectives and Conclusion
Alpha1-antitrypsin deficiency (AATD) Analyst Views
Alpha1-antitrypsin deficiency (AATD) Key Companies
AppendixList of Tables
Table 1 Total Products for Alpha1-antitrypsin deficiency (AATD)
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 ProductsList of Figures
Figure 1 Total Products for Alpha1-antitrypsin deficiency (AATD)
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
Companies Mentioned
A selection of companies mentioned in this report includes:
- Alnylam Pharmaceuticals
- Arrowhead Pharmaceuticals
- Kamada
- Mereo BioPharma
- Santhera Pharmaceuticals
- Centessa Pharmaceuticals
- Inhibrx
- WaVe Life Sciences
- Promethera Biosciences
- Beam Therapeutics
