Vaso-occlusive crisis-associated with sickle cell disease - Pipeline Insight, 2024

This “Vaso-occlusive-crisis-associated-with-sickle-cell-disease - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Vaso-occlusive-crisis-associated-with-sickle-cell-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

Vaso-occlusive-crisis-associated-with-sickle-cell-disease Understanding

Vaso-occlusive-crisis-associated-with-sickle-cell-disease: Overview

A vaso-occlusive crisis, or VOC, occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen. This in turn sets in motion an inflammatory response as the body tries to rectify the problem. The result is substantial pain, which can affect any part of the body, but most commonly occurs in the back, chest, or extremities. In most cases, it is not known what triggers a VOC. However, some factors are known to increase the likelihood of these pain crises, such as cold temperatures, dehydration, infection, and stress. VOCs are sometimes described as occurring in four phases: the first phase, which lasts around three days, is associated with a low-intensity, aching pain that may be accompanied by tingling or numbness as the affected tissue starts to lose oxygen. In the second phase, aching rapidly increases to an acute pain as affected tissue starts to die. The third phase is characterized by the body’s inflammatory response to tissue death, with constant, severe pain that may be accompanied by fever and usually lasts several days. The fourth and final phase is the resolution of the crisis, which usually takes place over one to two days. VOCs can be extremely painful and are considered a medical emergency. Short-term or acute management largely focuses on controlling pain with pain-relieving medications (analgesics). Opioids are commonly used for this purpose.

Vaso-occlusive-crisis-associated-with-sickle-cell-disease - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Vaso-occlusive-crisis-associated-with-sickle-cell-disease pipeline landscape is provided which includes the disease overview and Vaso-occlusive-crisis-associated-with-sickle-cell-disease treatment guidelines. The assessment part of the report embraces, in depth Vaso-occlusive-crisis-associated-with-sickle-cell-disease 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, Vaso-occlusive-crisis-associated-with-sickle-cell-disease 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 Vaso-occlusive-crisis-associated-with-sickle-cell-disease.
• In the coming years, the Vaso-occlusive-crisis-associated-with-sickle-cell-disease 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 Vaso-occlusive-crisis-associated-with-sickle-cell-disease 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 Vaso-occlusive-crisis-associated-with-sickle-cell-disease treatment market. Several potential therapies for Vaso-occlusive-crisis-associated-with-sickle-cell-disease are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Vaso-occlusive-crisis-associated-with-sickle-cell-disease 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 Vaso-occlusive-crisis-associated-with-sickle-cell-disease) 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.

Vaso-occlusive-crisis-associated-with-sickle-cell-disease Emerging Drugs Chapters

This segment of the Vaso-occlusive-crisis-associated-with-sickle-cell-disease 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.

Vaso-occlusive-crisis-associated-with-sickle-cell-disease Emerging Drugs

Inclacumab: Global Blood Therapeutics Global Blood Therapeutics (GBT) is developing inclacumab - a novel, fully human monoclonal antibody P-selectin inhibitor - for SCD patients with frequent pain crises, also known as vaso-occlusive crises (VOCs). P-selectin inhibition is clinically validated to reduce VOCs, a complication of SCD. GBT has an exclusive worldwide licensing agreement with Roche for inclacumab, which has established pharmacokinetic data, safety and tolerability in more than 500 patients. We have initiated two pivotal Phase 3 studies evaluating the effect of inclacumab on reducing the frequency of VOCs and on hospital readmissions for people with SCD. The THRIVE (Therapy for Reduction with Inclacumab of VOC Episodes) Studies are currently recruiting.

L-citrulline: Asklepion Pharmaceuticals Intravenous L-citrulline, a non-essential amino acid is being developed by Asklepion, under a license from Vanderbilt University. Researchers at Vanderbilt University found that cardiopulmonary bypass significantly decreased blood levels of citrulline, which compromised the ability to make an important blood vessel dilator, nitric oxide, during surgery and in the postoperative period. Currently, it is in Phase I/II stage of clinical trial evaluation to treat Acute Vaso Occlusive Crisis (VOC).

Vaso-occlusive-crisis-associated-with-sickle-cell-disease: Therapeutic Assessment

This segment of the report provides insights about the different Vaso-occlusive-crisis-associated-with-sickle-cell-disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Vaso-occlusive-crisis-associated-with-sickle-cell-disease

There are approx. 5+ key companies which are developing the therapies for Vaso-occlusive-crisis-associated-with-sickle-cell-disease. The companies which have their Vaso-occlusive-crisis-associated-with-sickle-cell-disease drug candidates in the most advanced stage, i.e. phase III include, Global Blood Therapeutics.

Phases

This 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

Vaso-occlusive-crisis-associated-with-sickle-cell-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

• 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.

Vaso-occlusive-crisis-associated-with-sickle-cell-disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Vaso-occlusive-crisis-associated-with-sickle-cell-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 Vaso-occlusive-crisis-associated-with-sickle-cell-disease drugs.

Vaso-occlusive-crisis-associated-with-sickle-cell-disease Report Insights

• Vaso-occlusive-crisis-associated-with-sickle-cell-disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Global Blood Therapeutics
• Asklepion Pharmaceuticals, LLC
• Prolong Pharmaceuticals
• CSL Behring
• GlycoMimetics
• Hillhurst Pharmaceuticals

Key Products

• Inclacumab
• L-citrulline
• SANGUINATE
• CSL889
• GMI-1687
• HBI 002