Sickle Cell Disease- Pipeline Insight, 2024

DelveInsight's, "Sickle Cell Disease- Pipeline Insight, 2024" report provides comprehensive insights about 40+ companies and 50+ pipeline drugs in 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

Sickle Cell Disease: Understanding

Sickle Cell Disease: Overview

Sickle Cell Disease (SCD) is a group of inherited red blood cell disorders that affect hemoglobin, the protein that carries oxygen through the body. Healthy red blood cells are round, and they move through small blood vessels to carry oxygen to all parts of the body. In someone who has SCD, the hemoglobin is abnormal, which causes the red blood cells to become hard and sticky and look like a C-shaped farm tool called a "sickle." The sickle cells die early, which causes a constant shortage of red blood cells. Sickle cells can get stuck and block blood flow, causing pain and infections. Complications of sickle cell disease occur because the sickled cells block blood flow to specific organs. The sickle cells also block the flow of blood through vessels, resulting in lung tissue damage that causes acute chest syndrome, pain episodes, stroke, priapism and damage to the spleen, kidneys and liver.

Sickle cells are destroyed rapidly in the bodies of people with the disease, causing anemia which is why it is commonly known as sickle cell anemia. Sickle cell disease is a serious and lifelong condition, although long-term treatment can help manage many of the problems associated with it. The disease affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. There is an atypical hemoglobin molecule called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape. Sickle cell disease is caused by a mutation in the hemoglobin-Beta gene found on chromosome 11.

SCD can be prevented prenatally, diagnosed in utero or in the newborn period by screening, or be detected at any time during life. The type of testing done depends on the age of the patient. DNA testing can be used for prenatal diagnosis or to confirm a diagnosis of the sickle cell genotype. Screening of neonates is available in most US states and involves hemoglobin electrophoresis. Screening and diagnosis in children and adults involve examination of the peripheral smear, hemoglobin solubility testing, and hemoglobin electrophoresis. The gold standard for pain assessment and diagnosis is patient self-report. There are no reliable diagnostic tests to confirm the presence of acute or chronic pain in individuals with SCD except when there are identifiable causes like avascular necrosis on imaging or leg ulcers on exam.

Treatment for sickle cell anemia is usually aimed at avoiding crises, relieving symptoms and preventing complications. Treatments might include medications to reduce pain and prevent complications, and blood transfusions, as well as a bone marrow transplant. Researchers are exploring whether inserting a normal gene into the bone marrow of people with sickle cell anemia will result in normal hemoglobin along with exploring the possibility of turning off the defective gene while reactivating another gene responsible for the production of fetal hemoglobin.

"Sickle Cell Disease- Pipeline Insight, 2024" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Sickle Cell Disease pipeline landscape is provided which includes the disease overview and Sickle Cell Disease treatment guidelines. The assessment part of the report embraces, in depth 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, Sickle Cell Disease 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 Sickle Cell Disease R&D. The therapies under development are focused on novel approaches to treat/improve Sickle Cell Disease.

Sickle Cell Disease Emerging Drugs Chapters

This segment of the 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.

Sickle Cell Disease Emerging Drugs

• Inclacumab: Pfizer

Inclacumab, a fully human IgG4 anti-P-selectin monoclonal antibody, is being developed for the reduction of vaso-occlusive crises (VOCs) in patients with SCD. P-selectin is a protein that mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab has the potential to be a best-in-class option for reducing VOCs in people with SCD, with the potential for quarterly, rather than monthly dosing. The drug was being developed by the Global Blood Therapeutics (GBT), which is acquired by the Pfizer. GBT has exclusive worldwide rights to inclacumab as part of the company's licensing agreement with Roche. Inclacumab is in Phase III stage of the development for SCD.

• Mitapivat: Agios Pharmaceuticals

Mitapivat is an oral small molecule designed to activate the enzyme pyruvate kinase-R (PKR). This enzyme is important for glycolisis - the process of generating cellular energy by breaking down sugar molecules. When PKR is active, it uses up 2, 3-diphosphoglycerate (2, 3-DPG). In SCD, 2, 3-DPG is thought to accumulate in red blood cells and cause them to become misshapen by prompting hemoglobin molecules to clump together in a process known as polymerization. By activating PKR, Mitapivatis expected to lower the 2, 3-DPG accumulation and thereby lessen hemoglobin polymerization and red blood cell sickling. Agios Pharmaceuticals has initiated a Phase II/III trial called RISE UP to test its investigational therapy Mitapivat (AG-348) in people with sickle cell disease.

• EPI01 (NDec): Novo Nordisk A/S

EPI01 is an oral, fixed dose formulation of a DNA methyl-transferase enzyme 1 and cytidine deaminase inhibitor, decitabine and tetrahydrouridine, which has potential to work by increasing the amount of foetal haemoglobin that can substitute the defective haemoglobin in SCD patients, thereby intending to prevent the deformation of the red blood cells and improving the oxygen level in the blood. EpiDestiny has been granted Rare Pediatric Disease, Fast Track and Orphan Designations by the U.S. Food and Drug Administration (FDA) for EPI01. In April 2018, Novo Nordisk and EpiDestiny announced that Novo Nordisk has obtained an exclusive worldwide licence to EpiDestiny's sickle cell disease (SCD) programme, EPI01. As per this agreement, EpiDestiny is eligible to receive more than 400 million US dollars in upfront, development and sales milestone payments and will get royalties on net sales. Currently, the drug is in the Phase II stage of its development for the treatment of Sickle Cell Disease.

• GSK 4172239: GlaxoSmithKline

GSK 4172239 acts by inhibiting the DNA cytosine 5 methyltransferase 1 inhibitor. It is administered through oral route. Currently, the drug is in the Phase I stage of its development to test its investigational therapy in people with sickle cell disease

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

Sickle Cell Disease: Therapeutic Assessment

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

• Major Players in Sickle Cell Disease
• There are approx. 40+ key companies which are developing the therapies for Sickle Cell Disease. The companies which have their Sickle Cell Disease drug candidates in the most advanced stage, i.e. Phase III include, Pfizer.
• Phases

DelveInsight's report covers around 50+ 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

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

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

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 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 Sickle Cell Disease drugs.

Sickle Cell Disease Report Insights

• Sickle Cell Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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 Sickle Cell Disease drugs?
• How many 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 Sickle Cell Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the 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 Sickle Cell Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• CRISPR therapeutics
• Bluebird Bio
• Pfizer
• Novo Nordisk
• Agios Pharmaceuticals
• Alexion Pharmaceuticals
• Takeda
• Prolong Pharmaceuticals
• Roche
• Beam Therapeutics
• Editas Medicine
• Sangamo Therapeutics
• Bellicum Pharmaceuticals
• Invenux
• EpiDestiny
• Hillhurst Biopharmaceuticals
• CSL Behring
• Fulcrum Therapeutics
• Sana Biotechnology

Key Products

• CTX001
• LentiGlobin BB305
• Inclacumab
• Etavopivat
• Mitapivat
• ALXN1820
• TAK-755
• Sanguinate
• Crovalimab
• BEAM101
• EDIT 301
• BIVV003
• BPX-501
• SCD-101
• Nicotinamide
• HBI-002
• CSL889
• FTX-6058
• SG418