Angioedema - Pipeline Insight, 2024

This “Angioedema - Pipeline Insight, 2024,” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Angioedema 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

Angioedema Understanding

Angioedema: Overview

Angioedema is defined as subcutaneous tissues and/or submucosal tissues circumscribed non-pitting edema affecting lips, face, neck, and extremities oral cavity, larynx, and gut. It becomes life-threatening when it involves the larynx, while intestinal angioedema is painful and mimics acute abdomen. In inherited (hereditary angioedema) mutations in the gene encoding for C1-inhibitor cause hereditary angioedema and it is an autosomal dominant condition. Hereditary angioedema is a rare disease that often leads to delay in diagnosis as well as a misdiagnosis. Other factors leading to a misdiagnosis are unknown family history, gastrointestinal manifestations of disease without cutaneous involvement. Angioedema is caused by an increase in local capillary permeability and plasma extravasation, usually mediated by mast cells, histamine, or bradykinin release. Angioedema is most commonly histamine-mediated; mast cell and basophil stimulation results in histamine release. Angioedema with urticaria tends to suggest a histaminergic form, which includes acute allergic angioedema and histaminergic idiopathic angioedema. The exact cause depends on the type of angioedema a patient has.

Angioedema - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Angioedema pipeline landscape is provided which includes the disease overview and Angioedema treatment guidelines. The assessment part of the report embraces, in depth Angioedema 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, Angioedema collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Angioedema.
• In the coming years, the Angioedema market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

The companies and academics that are working to assess challenges and seek opportunities that could influence Angioedema R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.

• A detailed portfolio of major pharma players who are involved in fueling the Angioedema treatment market. Several potential therapies for Angioedema are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Angioedema market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Angioedema) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Angioedema Emerging Drugs Chapters

This segment of the Angioedema report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Angioedema Emerging Drugs

NTLA-2002: Intellia Therapeutics NTLA-2002 is Intellia’s wholly owned development candidate for the treatment of HAE and is company’s second in vivo knockout therapeutic candidate. NTLA-2002 is currently being studied in a first-in-human Phase 1/2 clinical trial Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellia’s second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which together carry out the precision editing.

Donidalorsen: Ionis Pharmaceuticals Donidalorsen, formerly known as IONIS-PKK-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of prekallikrein, or PKK, to treat patients with hereditary angioedema, or HAE. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. HAE is a rare genetic disease characterized by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea. HAE can be fatal if swelling occurs in the larynx. In patients with frequent or severe attacks, doctors may use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks. However, current prophylactic treatment approaches are very limited and have major tolerability issues due to challenging administration requirements leaving patients with few therapeutic options.

Angioedema: Therapeutic Assessment

This segment of the report provides insights about the different Angioedema drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Angioedema

There are approx. 20+ key companies which are developing the therapies for Angioedema. The companies which have their Angioedema drug candidates in the most advanced stage, i.e. phase III include, Ionis Pharmaceuticals.

Phases

This report covers around 20+ 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

Angioedema pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

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

Angioedema: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Angioedema 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 Angioedema drugs.

Angioedema Report Insights

• Angioedema Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Angioedema 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 Angioedema drugs?
• How many Angioedema drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Angioedema?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Angioedema 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 Angioedema and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Intellia Therapeutics
• Ionis Pharmaceuticals
• CSL Behring
• Kal VistaPharmaceuticals
• Bio MarinPharmaceutical
• Pharvaris Netherlands B.V.
• Pharming Group NV
• Attune Pharmaceuticals
• Astria Therapeutics
• Bridge Medicines
• CURACLE
• CAMP4 Therapeutics
• Orchard Therapeutics

Key Products

• NTLA-2002
• Donidalorsen
• CSL312
• KVD900
• BMN 331
• PHA-022121
• OTL 105
• ATN 249
• STAR-0215
• CU 06
• Garadacimab

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