Sickle Cell Disease Competitive landscape

DelveInsight’s, “Sickle Cell Disease Competitive landscape 2026” report provides comprehensive insights about 40+ companies and 50+ drugs in Sickle Cell Disease (SCD)  Competitive landscape. It 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 (SCD) Understanding

Sickle Cell Disease (SCD) Overview

Sickle cell disease (SCD) refers to a group of inherited red blood cell conditions caused by a mutation in the HBB gene on chromosome 11, where valine replaces glutamic acid at the sixth position of the beta-globin chain. This change produces hemoglobin S (HbS), which polymerizes under low oxygen conditions, causing red blood cells to become rigid and sickle-shaped. These distorted cells lead to blocked blood flow (vaso-occlusion), ongoing red cell destruction, and damage to multiple organs, resulting in symptoms such as severe pain episodes, anemia, increased stroke risk, and complications like acute chest syndrome. SCD is most prevalent among individuals of African, Mediterranean, Middle Eastern, and Indian ancestry, partly due to the protective advantage of the carrier state against malaria. It affects millions worldwide and includes forms such as sickle cell anemia (HbSS), HbSC disease, beta-thalassemia, and the generally asymptomatic carrier state (HbAS). 

The pathophysiology of sickle cell anemia is mainly driven by hemolysis and vaso-occlusion. A mutation in the beta-globin gene produces hemoglobin S (HbS), which polymerizes in low oxygen conditions, causing red blood cells to become rigid and sickle-shaped—initially reversible but eventually permanent, increasing cell breakdown and vessel blockage. Factors such as low oxygen affinity of HbS, high 2,3-DPG levels, red cell dehydration, and membrane damage promote this process. Oxidative stress further damages cells, releasing free hemoglobin and arginase-1, which reduce nitric oxide and impair vascular function. Additionally, interactions between sickled cells, endothelium, and blood components like neutrophils and platelets enhance inflammation, adhesion, and thrombosis, leading to vaso-occlusion and tissue injury. 

The diagnosis of sickle cell disease is done using newborn screening, blood tests, and confirmatory hemoglobin or genetic analysis. Newborns are routinely screened via heel-prick blood samples to detect abnormal hemoglobins (HbS, HbC, HbF). Positive results are followed by tests such as complete blood count (CBC), sickle cell solubility testing, and hemoglobin electrophoresis or high-performance liquid chromatography (HPLC) to identify and quantify variants (e.g., HbSS, HbSC, HbS/β-thalassemia). DNA testing may be used in complex cases.

In older individuals, diagnosis is often prompted by symptoms like pain crises or anemia and confirmed with similar tests. Early detection enables timely interventions, including counseling, vaccination, preventive antibiotics, and hydroxyurea therapy to reduce complications. 

Treatment aims to relieve symptoms, prevent complications, and alter the course of the disease. Hydroxyurea remains the cornerstone therapy, as it raises fetal hemoglobin levels and reduces both pain episodes and hemolysis. Emerging treatments such as voxelotor, which limits HbS polymerization; crizanlizumab, which blocks cell adhesion to prevent vaso-occlusion; and L-glutamine, which decreases oxidative stress offer additional benefits. Supportive measures include blood transfusions, infection prevention, and management of complications. In certain patients, hematopoietic stem cell transplantation can provide a potential cure, although its use is limited. 

Discover the latest Sickle Cell Disease Competitive landscape, emerging therapies, and clinical advancements shaping the future of treatment in 2026.

Sickle Cell Disease Pipeline Report Highlights

• In March 2026, EpiFrontier Therapeutics, Inc., a biotechnology company developing transformative therapies for beta globin disorders, announced its award of up to USD 32 million in non-dilutive funding from the Japan Agency for Medical Research and Development (AMED). The grant will advance the clinical development of EPF-001, a novel small molecule G9a inhibitor designed to increase fetal hemoglobin expression in patients with sickle cell disease.
• In March 2026, Beam Therapeutics announced a USD 500 million strategic financing agreement with Sixth Street, including USD 100 million upfront and up to USD 400 million tied to milestones. The funding supports commercialization of its sickle cell therapy risto-cel while providing non-dilutive capital and extending its financial runway.
• In February 2026, Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, reported positive 12-week results from the 20 mg dose cohort of the Phase Ib PIONEER trial of pociredir in sickle cell disease.
• In February 2026, Zydus Lifesciences announced that The US FDA has granted Orphan Drug Designation to Zydus Lifesciences’ sickle cell therapy, Desidustat, an oral therapy being explored for its potential to address underlying disease mechanisms, not just symptoms. 
• In December 2025, Beam Therapeutics Inc., a biotechnology company, announced new safety and efficacy data from its BEACON Phase I/II clinical trial of ristoglogene autogetemcel (risto-cel), formerly known as BEAM-101, an investigational genetically modified ex vivo base editing cell therapy, in patients with sickle cell disease with severe vaso-occlusive crises (VOCs).
• In November 2025, Agios Pharmaceuticals, Inc. a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, announced topline results from the 52-week double-blind period of the global RISE UP Phase III trial of mitapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years or older with sickle cell disease
• In July 2025, Hillhurst Biopharmaceuticals announced the initiation of a Phase IIa clinical trial with the first patient dosed for HBI-002, an oral low-dose carbon monoxide therapy for sickle cell disease. The trial aims to evaluate the safety, efficacy, and pharmacokinetics of escalating doses of HBI-002 in both adolescent and adult patients. The primary objective is to assess the frequency and severity of treatment-related adverse events, while secondary endpoints include measuring peak hemoglobin levels and changes in key blood parameters such as hemoglobin, hematocrit, and cell counts. Insights from this study will guide the design of a larger Phase IIb trial planned for 2026.
• In June 2025, Sanofi announced that the US Food and Drug Administration (FDA) has granted Orphan drug designation to Wayrilz for Sickle Cell disease.

Sickle Cell Disease Marketed Drugs

Company Overview: Emmaus Medical, Inc.

Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. Our initial efforts have focused on treatments for Sickle Cell Disease, a genetic disorder. The aim of Emmaus Life Sciences is to improve the lives of people in need through the discovery, development and commercialization of innovative treatments and therapies. The focus of the company has been on rare and orphan diseases, such as sickle cell disease. The company is now extending our efforts to include conditions and diseases affecting larger populations, such as diverticulosis.
Product Description: Endari

Endari (L-glutamine oral powder), indicated to reduce the acute complications of sickle cell disease in adults and children 5 years and older, is approved for marketing in the United States, Israel, Kuwait, Qatar, the United Arab Emirates, Bahrain and Oman. The mechanism of action of the amino acid L-glutamine in treating sickle cell disease is not fully understood. Oxidative stress phenomena are involved in the pathophysiology of SCD. Sickle red blood cells (RBCs) are more susceptible to oxidative damage than normal RBCs, which may contribute to the chronic hemolysis and vaso-occlusive events associated with SCD.

Company Overview: Novartis Pharmaceuticals

Novartis Pharmaceuticals is reimagining medicine to improve and extend people's lives. As a leading global medicines company, the company use innovative science and digital technologies to create transformative treatments in areas of great medical need. In the quest to find new medicines, the company consistently rank among the world's top companies investing in research and development. Founded in 1996 the company Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. 
Product Description: Adakveo

Adakveo (crizanlizumab-tmca) – previously known as SEG101 – is indicated to reduce the frequency of VOCs, or pain crises, in adults and pediatric patients aged 16 years and older with sickle cell disease. The drug is a humanized IgG2 kappa monoclonal antibody that binds to P-selectin and blocks interactions with its ligands, including P-selectin glycoprotein ligand 1 (PSGL-1). Crizanlizumab can also dissociate preformed Pselectin/PSGL-1 complex. Binding P-selectin on the surface of the activated endothelium and platelets blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes. Crizanlizumab-tmca is produced using recombinant DNA technology in Chinese hamster ovary (CHO) cells. It is composed of 2 heavy chains, each containing 448 amino acids, and 2 light chains each containing 218 amino acids, with a theoretical molecular weight of approximately 146 kDa. The drug is supplied as a sterile, preservative-free, clear to opalescent, colorless to slightly brownish-yellow solution for dilution and subsequent administration by intravenous infusion. Each 10 mL vial contains 100 mg crizanlizumab-tmca, citric acid (5.4 mg), polysorbate 80 (2 mg), sodium citrate (50.5 mg), sucrose (753.3 mg) and water for injection with a pH of 6.

Sickle Cell Disease Emerging Drugs

Company Overview: Sanofi

Sanofi is an R&D driven, AI-powered Biopharma Company committed to improving people’s lives and delivering compelling growth. The company apply deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Founded in 1973, Sanofi have a major aim to chase the miracles of science to improve people’s lives, they drive progress and deliver positive impact for people and the communities, by addressing the most urgent healthcare, environmental, and societal challenges. The company aims to want to build a healthier, more resilient world. The company’s vision is to turn the impossible into the possible by discovering, developing, and delivering medicines and vaccines for millions of people around the world.

Product Description: Wayrilz (Rilzabrutinib)

Wayrilz (Rilzabrutinib) is an investigational oral therapy for sickle cell disease that works by inhibiting Bruton’s tyrosine kinase (BTK), a key regulator of immune and inflammatory responses. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in multiple immune-mediated disease processes and inflammatory pathways. Unlike treatments that directly target hemoglobin, rilzabrutinib addresses the underlying inflammation and vascular dysfunction associated with the disease. By blocking BTK activity, it reduces activation of immune cells and decreases the adhesion of blood cells to the endothelium, thereby helping to prevent vaso-occlusion—the primary cause of painful crises in sickle cell disease. The drug is currently in Phase III stage of development for the treatment of patients with Sickle Cell Disease.

Company Overview: Novo Nordisk A/S

Novo Nordisk is a leading global healthcare company founded in 1923 in Denmark, dedicated to defeating serious chronic diseases, primarily diabetes and obesity, rare diseases. This company is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for more than 100 years. The company have experience and capabilities that enable the company to drive change to help people defeat other serious chronic diseases such as obesity, rare blood, and endocrine disorders. With a US presence spanning 40 years, Novo Nordisk US is headquartered in New Jersey and employs approximately 10,000 people throughout the country across more than 10 manufacturing, R&D and corporate locations in seven states plus Washington DC.

Product Description: Etavopivat

Etavopivat is an investigational, oral, small molecule activator of erythrocyte pyruvate kinase (PKR) in development for the treatment of sickle cell disease (SCD) and other hemoglobinopathies. PKR activation is proposed to ameliorate the sickling of SCD red blood cells (RBCs) through multiple mechanisms, including reduction of 2,3-diphosphoglycerate (2,3-DPG), which consequently increases hemoglobin (Hb)-oxygen affinity; increased binding of oxygen reduces sickle hemoglobin polymerization and sickling. In addition, PKR activation increases adenosine triphosphate (ATP) produced via glycolytic flux, which helps preserve membrane integrity and RBC deformability. The drug is currently in Phase III stage of development for the treatment of patients with Sickle Cell Disease.

 Company Overview: Fulcrum Therapeutics

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Founded in2015, Fulcrum Therapeutics have a vision to develop small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s lead clinical program is pociredir, a small molecule designed to increase expression of HbF for the treatment of SCD. Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. 

Product Description: Pociredir

Pociredir, formerly known as FTX-6058, is a novel oral HbF inducer in development for treating SCD. It targets the embryonic ectoderm development (EED) subunit of polycomb repressive complex 2 (PRC2), inhibiting trimethylation of lysine 27 on histone H3 (H3K27me3) and leading to upregulation of HBG mRNA expression and thereby induction of HbF in red blood cells. Pociredir has been granted Fast Track and Orphan Drug Designation from the FDA for the treatment of Sickle Cell Disease. The drug is currently in Phase II stage of development for the treatment of patients with Sickle Cell Disease.

Company Overview: Hillhurst Biopharmaceuticals, Inc.

Hillhurst Biopharmaceuticals is a clinical-stage company focused on its proprietary GLASS™ platform, which enables the development of novel drug products based on known inhaled therapeutics . The platform overcomes limitations of traditional inhaled delivery methods. With the GLASS platform, the company create liquid drug products containing appropriate concentrations of the targeted medical agent, with uptake through the gastrointestinal system. Combining known medical gases with proprietary technology, the company produce novel liquid medicine. Hillhurst Biopharmaceuticals aims to liberate Sickle Cell Disease patients from the horrendous, recurrent, lifelong severe painful episodes and accompanying organ injury of this life limiting disease. 

Product Description: HBI-002

HBI-002 is a liquid drug product containing carbon monoxide (CO) intended for oral administration with target peak CO-hemoglobin (COHb) levels not exceeding 10%. CO has dual mechanisms of action including anti-sickling and anti-inflammatory processes. HBI-002 delivers low-dose carbon monoxide that inhibits hemoglobin S polymerization, improves oxygen binding, and reduces inflammation, thereby preventing red blood cell sickling and vaso-occlusion. A Phase I clinical study in healthy subjects under an Investigational New Drug (IND) application has been successfully completed. The drug is currently in Phase IIa stage of development for the treatment of patients with Sickle Cell Disease.

Company Overview: Novartis Pharmaceuticals

Novartis Pharmaceuticals is reimagining medicine to improve and extend people's lives. As a leading global medicines company, the company use innovative science and digital technologies to create transformative treatments in areas of great medical need. In the quest to find new medicines, the company consistently rank among the world's top companies investing in research and development. Founded in 1996 the company Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. 

Product Description: ITU512

ITU512 is an investigational, oral, low molecular weight (LMW) compound. It is designed as a molecular glue degrader targeting the WIZ transcription factor, a repressor of HbF production. By promoting degradation of WIZ, the drug reactivates HbF expression, thereby reducing red blood cell sickling and improving oxygen-carrying capacity which helps in treating sickle cell diseases. The drug is currently being evaluated in a global Phase I/II clinical trial to assess its safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in both healthy individuals and patients with sickle cell disease. 

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

Sickle Cell Disease (SCD) Analytical Perspective by DelveInsight

In-depth Commercial Assessment: Sickle Cell Disease (SCD) Collaboration Analysis by Companies
The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition – deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

Sickle Cell Disease (SCD) Competitive Landscape 

The report comprises a comparative assessment of Companies (by therapy, development stage, and technology).

Sickle Cell Disease (SCD) Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Sickle Cell Disease Market Unmet Needs

Discover actionable insights into the Sickle Cell Disease market trends, epidemiology trends, and forecast through 2036 to stay ahead in emerging therapies.

Key Questions Answered In The Sickle Cell Disease Pipeline Report

• Current Treatment Scenario and Emerging Therapies:
• How many Sickle Cell Disease companies are developing Sickle Cell Disease (SCD) drugs?
• How many Sickle Cell Disease (SCD) drugs are developed by each company?
• How many Sickle Cell Disease emerging drugs are in mid-stage, and late-stage of development for the treatment of Sickle Cell Disease (SCD)?
• What are the Sickle Cell Disease key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Sickle Cell Disease (SCD) 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 (SCD) and their status?
• What are the key designations that have been granted to the emerging and approved drugs?
   

Sickle Cell Disease Key Players

• Novartis Pharmaceuticals
• Pfizer
• Novo Nordisk 
• Sanofi
• Emmaus Medical, Inc.
• Agios Pharmaceuticals, Inc. 
• Fulcrum Therapeutics 
• Vertex Pharmaceuticals Incoporated
• Mast Therapeutics 
• Beam Therapeutics
• Afimmune 
• Sangamo Therapeutics
• Takeda 
• Theravia 
• Bristol-Myers Squibb 
• Genetix Biotherapeutics
• AstraZeneca
• CRISPR Therapeutics
• Prolong Therapeutics
• Hillhurst Biopharmaceuticals, Inc.

Sickle Cell Disease Key Products

• Endari 
• Adakveo 
• Wayrilz 
• Pociredir
• ITU512
• Etavopivat
• HBI-002
• PCV21 
• Mitapivat
• CTX001
• Risto-cel
• Epeleuton
• ST 400
• TAK-755
•  Hydroxycarbamide
• BMS-986470 
• bb1111
• Exa-cel
• Sanguinate
• Nula-Cel

Explore comprehensive insights into Sickle Cell Disease epidemiology trends, patient population forecasts, and growth opportunities through 2034 for strategic decision-making.