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Sickle Cell Disease - Pipeline Insight, 2020

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    Clinical Trials

  • 100 Pages
  • July 2020
  • Region: Global
  • DelveInsight
  • ID: 4471030

The “Sickle Cell Disease (SCD) – Pipeline Insight, 2020” report provides comprehensive insights about 45+ 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 is the name given for a group of inherited conditions that affect the red blood cells and the most serious type is called Sickle cell anemia. 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. 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 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.


The main symptoms of sickle cell disease include:

Sickle cell crises, these are painful episodes which can be very severe and can last up to a week

  • Increased risk of serious infections
  • Anemia (where red blood cells can't carry enough oxygen around the body), which can cause tiredness and shortness of breath
  • Some people also experience other problems such as delayed growth, strokes and lung problems

Early Signs and Symptoms

Some children with Sickle cell disease will start to have problems early on, and some later. Early symptoms may include:

  • Painful swelling of the hands and feet, known as dactylitis
  • Fatigue or fussiness from anemia
  • A yellowish color of the skin, known as jaundice, or whites of the eyes, known as icteris, that occurs when a large number of red cells hemolyze

The signs and symptoms of SCD will vary from person to person and can change over time.


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.

  • Medications: Medications used to treat sickle cell anemia include Antibiotics, Pain-relieving medications and Hydroxyurea (Droxia, Hydrea).
  • Immunizations: People with SCD should receive all recommended childhood vaccines. They should also receive additional vaccines to prevent other infections such as Pneumococcus, Influenza, etc.
  • Blood transfusions: In children with sickle cell anemia at high risk of stroke, regular blood transfusions can decrease the risk. Transfusions can also be used to treat other complications of sickle cell anemia, or they can be given to prevent complications.
  • Bone marrow transplant: This procedure usually uses a matched donor, such as a sibling, who doesn't have sickle cell anemia. If a donor is found, the person with sickle cell anemia receives radiation or chemotherapy to destroy or reduce his or her bone marrow stem cells. Healthy stem cells from the donor are injected intravenously into the bloodstream of the person with sickle cell anemia, where they migrate to the bone marrow and begin generating new blood cells.

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 III, II, I and preclinical. 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

Innohep: LEO Pharma

Innohep (Tinzaparin Sodium) is a registered trademark of LEO Pharmaceutical and is under development in Phase III stage of development for the treatment of Sickle Cell Disease. Tinzaparin is a low molecular weight heparin (LMWH), produced by enzymatic depolymerization of unfractionated heparin from porcine intestinal mucosa. It is a heterogeneous mixture of with an average molecular weight between 5500 and 7500 daltons. Tinzaparin is composed of molecules with and without a special site for high affinity binding to antithrombin III (ATIII). This complex greatly accelerates the inhibition of factor Xa. It is an anticoagulant and considered an antithrombotic. Tinzaparin must be given either subcutaneously or parenterally. LMWHs are less effective at inactivating factor IIa due to their shorter length compared to unfractionated heparin.

Rivipansel: Pfizer

Pfizer is developing Rivipansel, in partnership with Glycomimetics which is designed to relieve Vaso-occlusive crisis (VOC) of sickle cell disease. Rivipansel can treat VOC by reducing the cell adhesion, activation and inflammation that are believed to contribute to reducing blood flow through the microvasculature. It has been evaluated that this may reduce the pain experienced by individuals during sickle cell crisis and shorten their time in the hospital. Pfizer, is also studying whether this may reduce other medical problems that result from cell adhesion and activation, and vascular occlusion. The drug is in Phase III stage for the treatment of Sickle Cell Disease and Phase I stage of development for the treatment of Moderate to Normal Hepatic Impairment and Renal Impairment. The mode of administration is intravenous.

BPX-501 T cells: Bellicum Pharmaceuticals

BPX-501 T cells (also known as Rivo-cel) is being developed by Bellicum Pharmaceuticals. BPX-501 T cells are derived from non-mobilized donor leukapheresis and are expanded, transduced and selected for the iC9 gene, cryopreserved and shipped back to the clinical site. Currently it is in Phase I/II stage of development for the treatment of Hematological Disorders. Rivo-cel is a T cell therapy incorporating CaspaCIDe safety switch intended to improve HSCT outcomes in the treatment of hematological malignancies and inherited blood disorders. Rivo-cel is designed to treat immunodeficiency following allogeneic HSCT, preventing morbidity and mortality due to disease relapse and infection.

Canakinumab: Novartis

Novartis is developing Canakinumab (ACZ885), a selective, high-affinity, fully human monoclonal antibody that acts as an interleukin-1β (IL-1β) blocker in Phase II stage pf development for the treatment of Sickle Cell Anemia. IL-1β is a key cytokine in the inflammatory pathway known to drive the continued progression of inflammatory atherosclerosis. ACZ885 acts by blocking the action of IL-1β for a sustained period of time, therefore inhibiting inflammation that is caused by its over-production. Canakinumab is already approved under the brand name Ilaris for the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) including Familial Cold Auto inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS). Novartis is also developing ACZ885 for preventing cardiovascular risk. Company believe that ACZ885 is the first and only agent which has shown that selectively targeting inflammation significantly reduces cardiovascular risk in patients who have had a prior heart attack and have an increased cardiovascular inflammatory burden. Canakinumab is administered subcutaneously.

EPI01: Novo Nordisk

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.

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. 45+ 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, LEO Pharma, Pfizer, Merck Sharp & Dohme, Micelle BioPharma and AstraZeneca.


This report covers around 50+ products under different phases of clinical development like

  • Late stage products (Pre-registration and Phase III)
  • Mid-stage products (Phase II and Phase I/II) and
  • Early-stage products (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

  • Intravenous
  • Intradermal
  • Oral
  • Intramuscular
  • Subcutaneous etc.
  • Mechanism of Action

Products have been categorized under various MOAs such as

  • 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 modulators
  • LRF & NuRD modulators
  • Endothelin A receptor antagonist
  • Cytidine deaminase inhibitors
  • pyruvate kinas 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 Type

Products have been categorized under various Molecule types such as:

  • Antibody Drug Conjugate
  • Protein
  • Small Molecule
  • Protein
  • Peptide
  • Cell Therapy
  • Fatty Acid
  • Gene therapy
  • Recombinant proteins
  • Monoclonal Antibody
  • Stem cell therapy
  • Cell 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 III, II, I and preclinical 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.

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.
  • A better understanding of disease pathogenesis will also contribute to the development of Sickle cell disease (SCD) therapeutics.
  • In September 2013, GlycoMimetics announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation for GMI-1070 (rivipansel sodium) for the treatment of vaso-occlusive crisis (VOC) in patients with Sickle Cell Disease. It has received Fast Track Designation from the US Food and Drug Administration, or FDA, as well as Orphan Drug Designation from the FDA.
  • In February 2020, GlycoMimetics received notice for termination of its license agreement signed in October 2011 from Pfizer due to failure of its P-III RESET study to achieve 1EPs & 2EPs in patients with SCD experiencing VOC aged ≥6yrs. On termination, Pfizer loses rights to develop & commercialize licensed therapies including rivipansel and grants a non-exclusive license to GlycoMimetics to utilize Pfizer's IP, developed in connection with licensed therapies. In 2011, Pfizer and GlycoMimetics entered into a worldwide license agreement for the development and, if approved by applicable regulatory authorities, commercialization of rivipansel.
  • In April 2018, Sancilio Pharmaceuticals announced that it has received the European Medicines Agency's (EMA) Orphan Designation for its SC411 (which it plans to market as Altemia), a proposed treatment for sickle cell disease (SCD) in pediatric patients, in the European Union.
  • In July 2019, Novartis announced that, the US Food and Drug Administration (FDA) accepted the company's Biologics License Application (BLA) and has granted Priority Review for its investigational sickle cell medicine crizanlizumab (SEG101).
  • In January 2020, Aruvant announced that the US Food and Drug Administration has granted Orphan Drug designation to ARU-1801, Aruvant's investigational therapy for the treatment of sickle cell disease. The US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to ARU-1801.
  • Mozobil (plerixafor) was initially developed by Genzyme. In April 2011, Sanofi acquired Genzyme and it turned into a wholly owned subsidiary

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?

Table of Contents

IntroductionExecutive Summary
Sickle cell disease: 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

Sickle cell disease – Analytical Perspective
  • In-depth Commercial Assessment
  • Sickle cell disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Sickle cell disease Collaboration Deals
  • Company-Company Collaborations (Licensing/Partnering) Analysis
  • Company-University Collaborations (Licensing/Partnering) Analysis

Late Stage Products
  • Comparative Analysis

Innohep: LEO Pharm
  • Product Description
  • Research and Development
  • Product Development Activities

Rivipansel: Pfizer
  • Product Description
  • Research and Development
  • Product Development Activities
  • Drug profiles in the detailed report.
  • Mid Stage Products
  • Comparative Analysis

BPX-501 T cells: Bellicum Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities

Canakinumab: Novartis
  • Product Description
  • Research and Development
  • Product Development Activities
  • Drug profiles in the detailed report.

Early Stage Products
  • Comparative Analysis

Ambrisentan: Gilead Sciences
  • Product Description
  • Research and Development
  • Product Development Activities

BCL 11a shRNA (miR): Bluebird bio
  • Product Description
  • Research and Development
  • Product Development Activities
  • Drug profiles in the detailed report.

Pre-clinical and Discovery Stage Products
  • Comparative Analysis

FTX-6058: Fulcrum Therapeutics
  • Product Description
  • Research and Development
  • Product Development Activities
  • Drug profiles in the detailed report.

Inactive Products
  • Comparative Analysis

Sickle cell disease Key CompaniesSickle cell disease Key ProductsSickle cell disease- Unmet NeedsSickle cell disease- Market Drivers and BarriersSickle cell disease- Future Perspectives and ConclusionSickle cell disease Analyst ViewsSickle cell disease Key CompaniesAppendix
List of Tables
Table 1 Total Products for Sickle cell disease
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 Products
List of Figures
Figure 1 Total Products for Sickle cell disease
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:

  • CSL Behring
  • Magenta Therapeutics
  • Molecules for Health
  • Hillhurst Biopharmaceuticals
  • ORYZON therapeutics
  • Regenacy Pharmaceuticals
  • Dystrogen Therapeutics
  • ReveraGen
  • Editas Medicine
  • Bioverativ ( A Sanofi company)
  • Genethon
  • Syros Therapeutics
  • Gilead Sciences
  • Bluebird bio
  • CRISPR Therapeutics
  • Novo Nordisk
  • Forma Therapeutics
  • La Jolla
  • Merganser Biotech
  • NuvOx Pharma
  • Pfizer
  • Sanofi
  • LEO Pharma
  • Invenux
  • Micelle BioPharma
  • AstraZeneca
  • Merck Sharp & Dohme
  • Angiocrine Bioscience
  • Beam Therapeutics
  • Fulcrum Therapeutics
  • Nicox
  • Shire
  • TauTona Group
  • EpiDestiny
  • RadioRx
  • Bellicum Pharmaceuticals
  • Novartis
  • Miltenyi Biotec
  • Nova Laboratories
  • Gamida-Cell
  • Addmedica Sasa
  • Bristol Myers Squibb
  • Imara
  • Ironwood
  • ARUVANT (Roivant’s Vant)
  • Prolong Pharmaceuticals
  • Modus Therapeutics