Sickle Cell Disease Pipeline Insight

DelveInsight’s, “Sickle Cell Disease (SCD) – Pipeline Insights, 2023” 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 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.

 

"Sickle Cell Disease (SCD) - Pipeline Insight, 2023" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Sickle Cell Disease (SCD) pipeline landscape is provided which includes the disease overview and Sickle Cell Disease (SCD) treatment guidelines. The assessment part of the report embraces, in depth Sickle Cell Disease (SCD) 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 (SCD) 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 (SCD) R&D. The therapies under development are focused on novel approaches to treat/improve Sickle Cell Disease (SCD). 
  • This segment of the Sickle Cell Disease (SCD) 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 (SCD) Drugs

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.

 

CTX001: Vertex Pharmaceuticals

CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD. Currently it is being investigated in Phase III stage of development for SCD.

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

Sickle Cell Disease (SCD): Therapeutic Assessment

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

 

Major Players in Sickle Cell Disease (SCD)

There are approx. 40+ key companies which are developing the therapies for Sickle Cell Disease (SCD). The companies which have their Sickle Cell Disease (SCD) drug candidates in the most advanced stage, i.e. Phase III include Vertex Pharmaceuticals.

 

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

  • Intranasal
  • Intrathecal
  • Intravenous
  • Oral
  • Oral/Intravenous
  • Parenteral
  • Subcutaneous
  • Subcutaneous/Intramuscular
  • Transdermal

 

Molecule Type

Products have been categorized under various Molecule types such as

  • Antisense oligonucleotide
  • Gene therapy
  • Hormones
  • Neuropeptides
  • Oligonucleotides
  • Small Molecule
  • Triglyceride

 

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?

 

 

Introduction

Executive Summary

Sickle Cell Disease (SCD): 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 (SCD)– DelveInsight’s Analytical Perspective

Mid Stage Products (Phase III)

  • Comparative Analysis

CTX001: Vertex Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II/III)

  • Comparative Analysis

Mitapivat: Agios Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II)

  • Comparative Analysis

ARU-1801: Aruvant Sciences

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

GBT021601: Global Blood Therapeutics, Inc.

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical Stage Products

  • Comparative Analysis

TALGlobin01: Cellectis

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Sickle Cell Disease (SCD) Key Companies

Sickle Cell Disease (SCD) Key Products

Sickle Cell Disease (SCD)- Unmet Needs

Sickle Cell Disease (SCD)- Market Drivers and Barriers

Sickle Cell Disease (SCD)- Future Perspectives and Conclusion

Sickle Cell Disease (SCD) Analyst Views

Sickle Cell Disease (SCD) Key Companies

Appendix

List of Table

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

• Cellectis
• Sana Biotechnology
• Global Blood Therapeutics, Inc.
• CSL Behring
• Pfizer
• Aruvant Sciences
• Graphite Bio
• Novartis
• Agios Pharmaceuticals
• Forma therapeutics
• Vertex Pharmaceuticals
• Global Blood Therapeutics, Inc.
• Alfasigma
• Novo Nordisk
• bluebird bio
• ExCellThera
• Gamida Cell
• KM Biologics/Takeda
• Editas Medicine

 

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