Sickle Cell Disease Pipeline Landscape Expands With 50+ Drugs For Better Treatment Outlook.

Los Angeles, USA, May 11, 2021 (GLOBE NEWSWIRE) -- Sickle Cell Disease Pipeline Landscape Expands With 50+ Drugs For Better Treatment Outlook.

Approximately 40+ key companies are developing Sickle Cell Disease therapies. The companies with their Sickle cell disease drug candidates in the most advanced stage include Forma Therapeutics, Imara, Novartis, Aruvant Sciences and Novo Nordisk.

DelveInsight's "Sickle Cell Disease Pipeline Insight" report provides comprehensive insights about 40+ companies and 50+ pipeline drugs in the Sickle Cell Disease pipeline landscapes. It comprises Sickle Cell Disease pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Sickle Cell Disease therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Sickle Cell Disease pipeline products.     

Some of the key takeaways of the Sickle Cell Disease Pipeline Report  

• Key companies such as Syros Pharmaceuticals, Sana Biotechnology, CSL Behring, Novartis, Pfizer, Vertex Pharmaceuticals, Sangamo Therapeutics, Graphite Bio, Vifor Pharma, Imara Inc, and others are developing potential drug candidates to boost the Sickle Cell Disease treatment scenario. 
• In March 2021, Sangamo Therapeutics announced that the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle cell disease in the Phase 1/2 PRECIZN-1 study partnered with Sanofi. 
• In February 2021, Novartis announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD). 
• In December 2020, Editas Medicine announced preclinical data and successful development of a large-scale manufacturing process for EDIT-301, a potentially best-in-class, one-time, durable, autologous cell therapy medicine to treat sickle cell disease and beta-thalassemia. 
• In November 2020, CSL Behring announced that its investigational, plasma-derived hemopexin therapy (CSL889) received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of sickle cell disease (SCD).
• In April 2020, FORMA Therapeutics announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug designation for FORMA's lead investigational agent, FT-4202, currently in clinical development as a potentially disease-modifying treatment for sickle cell disease (SCD). 
• In January 2020, Aruvant announced that the FDA had granted Orphan Drug designation to ARU-1801, Aruvant's investigational therapy to treat Sickle Cell Disease. The US FDA granted Rare Pediatric Disease Designation to ARU-1801.

Get an overview of pipeline landscape @ Sickle Cell Disease Clinical Trials Analysis 

Sickle Cell Disease is a group of inherited red blood cell disorders that affects haemoglobin. Sickle cells are destroyed rapidly in the affected people's bodies, causing anaemia.

Sickle Cell Disease treatments might include medications to reduce pain and prevent complications, and blood transfusions, as well as a bone marrow transplant. 

Sickle Cell Disease Emerging Drugs

• FT-4202: Forma Therapeutics

Forma Therapeutics is developing FT-4202, an investigational agent designed to be a once-daily pill for the treatment of sickle cell disease. It is currently in the Phase II/III stage of development for sickle cell disease.  FT-4202 is a potent activator of pyruvate kinase-R (PKR), hypothesized to impact two pathways in the red blood cell. The clinical studies of FT-4202 will investigate whether decreasing 2,3-DPG may help oxygen bind to hemoglobin (i.e. increasing oxygen affinity), and thereby increase ATP and impact RBC function. The FDA has granted FT-4202 Fast Track, Rare Pediatric Disease and Orphan Drug designations. 

• IMR-687: Imara 

Imara is developing IMR-687, a highly selective and potent small molecule inhibitor of PDE9. PDE9 uniquely degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. Currently, it is in the Phase II stage of development for Hematological Disorders treatment. Lower levels of cGMP are often found in people with sickle cell disease and beta-thalassemia and are associated with impaired blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide-mediated vasodilation.
Blocking PDE9 acts to increase cGMP levels, which are associated with reactivation of fetal hemoglobin, or HbF, a natural hemoglobin produced during fetal development. Increased levels of HbF in red blood cells have been demonstrated to improve symptomology and lower disease burden in patients with sickle cell disease and patients with beta-thalassemia. 

• VIT-2763: Vifor Pharma

Vifor Pharma is developing VIT-2763  and is under development in the Phase II stage of development to treat Sickle Cell Disease.  It is the first oral ferroportin inhibitor with the potential for treating diseases with ineffective production of red blood cells and iron overload conditions, such as beta-thalassemia or Sickle Cell Disease (SCD). In January 2021, VIT-2763 was granted an orphan drug designation (ODD) from the US Food and Drug Administration (FDA) and a positive opinion from the European Medicines Agency (EMA) for the treatment of Sickle Cell Disease (SCD). VIT-2763 is an oral inhibitor of ferroportin, the only known mammalian iron exporter and essential for transport of iron from one cell type to another and therefore plays a key role in regulating iron uptake and distribution in the body. VIT-2763 binds to ferroportin and blocks it to prevent excessive iron release into the blood.

• Canakinumab: Novartis

Novartis is developing Canakinumab (ACZ885), a selective, high-affinity, fully human monoclonal antibody, which acts as an interleukin-1β (IL-1β) blocker in the Phase II stage of development for the Sickle Cell Anemia treatment. IL-1β is the main cytokine in the inflammatory pathway known to drive the persistent progression of inflammatory atherosclerosis. ACZ885 acts by inhibiting the action of IL-1β for a sustained period of time, therefore inhibiting inflammation caused by its over-production. Canakinumab is already authorized under the brand name Ilaris for the treatment of Cryopyrin Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS). Novartis is also making ACZ885 for inhibiting cardiovascular risk. Canakinumab is administered subcutaneously.

• EPI01: Novo Nordisk

EPI01 is an oral, fixed-dose formulation of a DNA methyltransferase enzyme 1 and cytidine deaminase inhibitor, decitabine and tetrahydrouridine, which has the potential to work by increasing the amount of foetal haemoglobin that can substitute the defective haemoglobin in Sickle Cell Disease patients, hence intending to stop the deformation of the red blood cells and enhancing 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.

For further information, refer to the detailed report @ Sickle Cell Disease Pipeline Therapeutics 

Scope of Sickle Cell Disease Pipeline Drug Insight    

• Coverage: Global 
• Major Players: 40+  Key Players
• Prominent Players: Syros Pharmaceuticals, Sana Biotechnology, CSL Behring, Novartis, Pfizer, Vertex Pharmaceuticals, Sangamo Therapeutics, Graphite Bio, Vifor Pharma, Imara Inc, and many others.   
• Key Drugs Profiles: 50+ Products
• Phases:  

• Sickle Cell Disease Therapies Late-stage (Phase III)  
• Sickle Cell Disease Therapies Mid-stage (Phase II)
• Sickle Cell Disease Therapies Early-stage (Phase I) 
• Sickle Cell Disease Pre-clinical stage and Discovery candidates   
• Discontinued and Inactive candidates 

• Mechanism of Action:

• Anti CD117 ADC
• Anti CD45 ADC
• Haemoglobin modulators
• Gene Replacement
• CXCR Agonist
• Lysine- specific demethylase 1 (LSD1) (KDM1A) inhibitor
• HDAC1, 2 Inhibitors
• NF-kappa B (NF-kB) inhibitor
• Heme oxygenase 1 modulator
• LRF & NuRD modulators
• Endothelin A receptor antagonist
• Cytidine deaminase inhibitors
• pyruvate kinase agonist
• Ferroportin Inhibitor
• Phosphodiesterase 9A Inhibitor
• CXC chemokine receptor 4 (CXCR4) Antagonist
• Factor Xa inhibitors; Thrombin inhibitors
• E-selectin inhibitors; L-selectin inhibitors; P-selectin inhibitors
• P2Y12 Receptor Antagonist
• Gene transference
• Cyclooxygenase inhibitors
• ADAM protein replacements
• Cytokine modulators
• Macrophage stimulants
• T lymphocyte replacements
• Interleukin-1 (IL-1) beta Inhibitor
• CD34 selected T-cell depleted allogeneic stem cells
• Fetal haemoglobin stimulants
• Phosphodiesterase 9A Inhibitors
• Guanylate Cyclase (sGC) stimulator
• Aryl hydrocarbon receptor (AHR) antagonist expanded stem cells
• Gene Transfer
• Oxygen carriers
• Adhesion Receptors

• Molecule Types:   

• Antibody-Drug Conjugate
• Protein 
• Small Molecule
• Peptide
• Fatty Acid
• Gene therapy
• Recombinant proteins
• Monoclonal Antibody
• Stem cell therapy
• Cell Therapy

• Route of Administration:

• Intravenous
• Intradermal
• Oral
• Intramuscular
• Subcutaneous  

• Product Types:

• Monotherapy
• Combination
• Mono/Combination 

Key Questions regarding Current Sickle Cell Disease Treatment Landscape and Emerging Therapies Answered in the Pipeline Report  

• What are the current options for Sickle Cell Disease treatment?
• How many companies are developing therapies for the treatment of Sickle Cell Disease? 
• How many are Sickle Cell Disease emerging therapies in the early-stage, mid-stage, and late development stages to treat Sickle Cell Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Sickle Cell Disease market? 
• Which are the dormant and discontinued products and the reasons for the same?
• What is the unmet need for current therapies for the treatment of Sickle Cell Disease?  
• What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Sickle Cell Disease therapies? 
• What are the critical designations that have been granted for the emerging therapies for Sickle Cell Disease? 
• How many patents are granted and pending for the emerging therapies to treat Sickle Cell Disease?   

Table of Contents

Get a customised pipeline report @ Sickle Cell Disease Drugs Pipeline Report  

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