Sickle Cell Disease Clinical Trial Pipeline Accelerates as 40+ Pharma Companies Rigorously Developing Drugs for the Market Entry

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Jul 11, 2023, 17:01 ET

The sickle cell disease pipeline and clinical trial analysis report deliver important insights on ongoing research, clinical strategies, upcoming therapeutics, and commercial analysis

LAS VEGAS, July 11, 2023 /PRNewswire/ -- DelveInsight's 'Sickle Cell Disease Competitive Landscape – 2023' report provides comprehensive global coverage of available, marketed, and pipeline sickle cell disease drugs in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space, company assessment, comparative assessment, and future growth potential of the sickle cell disease competitive domain .

Key Takeaways from the Sickle Cell Disease Competitive Landscape Report

Over 40+ sickle cell disease companies are evaluating 50+ sickle cell disease drugs in various stages of development, and their anticipated acceptance in the sickle cell disease market would significantly increase market revenue.
Key sickle cell disease companies such as 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, and others are evaluating new sickle cell disease drugs to improve the treatment landscape.
Promising sickle cell disease pipeline therapies such as 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, and others are under different phases of sickle cell disease clinical trials.
In May 2023, the US Food and Drug Administration (FDA) granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given as a one-time infusion for the treatment of sickle cell disease (SCD).
In April 2023, bluebird bio, Inc. announced the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs). The BLA includes a request for Priority Review, which, if granted, would shorten the FDA's review of the application to six months from the time of filing, versus a standard review timeline of 10 months. If approved, lovo-cel will be bluebird bio's third ex-vivo gene therapy approved by the FDA for a rare genetic disease and its second FDA approval for an inherited hemoglobin disorder, building on more than a decade of leadership in gene therapy.
In March 2023, BioLineRx Ltd. announced a collaboration with Washington University School of Medicine in St. Louis to advance a Phase I clinical trial that will evaluate the safety and feasibility of the Company's lead clinical candidate motixafortide to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in SCD.
In November 2022, C4X Discovery Holdings plc signed an exclusive worldwide licensing agreement with AstraZeneca worth up to $402 million, for its NRF2 Activator programme.
In October 2022, Pfizer announced the completion of its acquisition of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities starting with sickle cell disease (SCD).
In October 2022, Novo Nordisk completed the acquisition of Forma Therapeutics Holdings, Inc. Forma Therapeutics is a clinical-stage biopharmaceutical company focused on transforming the lives of patients with sickle cell disease (SCD) and rare blood disorders.
In June 2022, Precision BioSciences, Inc., a clinical-stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis Pharma AG.

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Sickle Cell Disease Overview

Sickle cell disease is a group of inherited red blood cell diseases that affect hemoglobin, the protein that transports oxygen throughout the body. Red blood cells in good health are spherical and flow through small blood channels to carry oxygen to all regions of the body. Sickle cell disease patients have defective hemoglobin, which causes red blood cells to become hard and sticky, resembling a C-shaped farm tool known as a "sickle." Sickle cell disease can be prevented during pregnancy, diagnosed in utero or during the newborn period, or found at any time during life. The sort of tests performed is determined by the patient's age. DNA testing can be utilized for prenatal diagnosis or to validate a sickle cell genotype diagnosis. Neonate screening is offered in the majority of US states and involves hemoglobin electrophoresis.

The goal of sickle cell anemia treatment is usually to avoid crises, relieve symptoms, and prevent consequences. Some medical treatments and home cures are available to help you feel better. Medication to relieve discomfort and prevent complications, blood transfusions, and a bone marrow transplant are all possible treatments. Researchers are investigating if putting a normal gene into the bone marrow of persons with sickle cell anemia can result in normal hemoglobin, as well as turning off the defective gene while reactivating another gene important for fetal hemoglobin production.

Find out more about sickle cell disease treatment drugs @ Drugs for Sickle Cell Disease Treatment

Sickle Cell Disease Pipeline Analysis: Drug Profile

Inclacumab: Pfizer

Inclacumab is a completely human IgG4 anti-P-selectin monoclonal antibody that is being developed to reduce vaso-occlusive crises (VOCs) in sickle cell disease patients. P-selectin is a protein that mediates cell adhesion and has been scientifically proven to alleviate discomfort caused by VOCs in persons with sickle cell disease. Preclinical data indicate that inclacumab has the potential to be a best-in-class alternative for lowering VOCs in persons with sickle cell disease, with quarterly rather than monthly treatment. Global Blood Therapeutics (GBT), which was acquired by Pfizer, created the medication. As part of its licensing arrangement with Roche, GBT owns exclusive worldwide rights to inclacumab. Inclacumab is currently in Phase III development for sickle cell disease.

Mitapivat: Agios Pharmaceuticals

Mitapivat is an orally administered small drug that activates the enzyme pyruvate kinase-R (PKR). This enzyme is required for glycolysis, which is the process of producing cellular energy by breaking down sugar molecules. When the PKR enzyme is activated, it consumes 2, 3-diphosphoglycerate (2, 3-DPG). In SCD, it is assumed that 2, 3-DPG accumulates in red blood cells, causing them to become malformed by causing hemoglobin molecules to clump together in a process known as polymerization. Mitapivatis is intended to reduce 2, 3-DPG accumulation and thereby hemoglobin polymerization and red blood cell sickling via activating PKR. Agios Pharmaceuticals has launched the RISE UP Phase II/III trial to evaluate the experimental treatment Mitapivat (AG-348) in people with sickle cell disease.

A snapshot of the Sickle Cell Disease Pipeline Drugs mentioned in the report:

Sickle Cell Disease Drugs	Company	Phase	MoA
Exagamglogene autotemcel	CRISPR Therapeutics/Vertex Pharmaceuticals	Preregistration	Cell replacements; Fetal haemoglobin expression stimulants
Inclacumab	Pfizer	Phase III	P selectin inhibitors
Mitapivat	Agios Pharmaceuticals	Phase II/III	Pyruvate kinase stimulants
Canakinumab	Novartis	Phase II	Interleukin 1 beta inhibitors
ALXN1820	Alexion Pharmaceuticals	Phase II	Properdin inhibitors
Crovalimab	Chugai Pharmaceutical/Roche	Phase II	Complement C5 inhibitors
EDIT 301	Editas Medicine	Phase I/II	Cell replacements; Gene transference
BIVV003	Sangamo Therapeutics	Phase I/II	Cell replacements; Gene transference; Haemoglobin stimulants
BEAM101	Beam Therapeutics	Phase I	Gene transference
Hemopexin	CSL Behring	Phase I	Hemopexin replacements

Learn more about the emerging sickle cell disease pipeline therapies @ Sickle Cell Disease Clinical Trials

Scope of the Sickle Cell Disease Competitive Landscape Report

Coverage: Global
Key Sickle Cell Disease Companies: 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, and others
Key Sickle Cell Disease Pipeline Therapies: 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, and others
Company Analysis, Therapeutic Assessment, Pipeline Assessment, Inactive drugs assessment, Unmet Needs

Dive deep into rich insights for new drugs for sickle cell disease treatment, visit @ Sickle Cell Disease Drugs

Table of Contents

1.	Sickle Cell Disease Pipeline Report Introduction
2.	Sickle Cell Disease Pipeline Report Executive Summary
3.	Sickle Cell Disease Pipeline: Overview
4.	Sickle Cell Disease Marketed Drugs
4.1.	ADAKVEO: Novartis
5.	Sickle Cell Disease Clinical Trial Therapeutics
6.	Sickle Cell Disease Pipeline: Late-Stage Products (Pre-registration)
7.	Sickle Cell Disease Pipeline: Late-Stage Products (Phase III)
7.1.	Inclacumab: Pfizer
8.	Sickle Cell Disease Pipeline: Mid-Stage Products (Phase II)
8.1.	Canakinumab: Novartis
9.	Sickle Cell Disease Pipeline: Early-Stage Products (Phase I)
9.1.	TAK-755: Takeda
10.	Sickle Cell Disease Pipeline Therapeutics Assessment
11.	Inactive Products in the Sickle Cell Disease Pipeline
12.	Company-University Collaborations (Licensing/Partnering) Analysis
13.	Unmet Needs
14.	Sickle Cell Disease Market Drivers and Barriers
15.	Appendix

For further information on the sickle cell disease pipeline therapeutics, reach out @ Sickle Cell Disease Treatment Drugs

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