sickle cell disease pipeline insight

DelveInsight’s, “Sickle Cell Disease Pipeline Insights, 2025,” report provides comprehensive insights about 55+ companies and 60+ drugs in Sickle Cell Disease  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: 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. The disease affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. The common symptoms associated with sickle cell disease are anemia, pain in the joints, acute chest syndrome, splenic sequestration (pooling), swelling of hands and feet, sickle-shaped red blood cells block blood circulation in the hands and feet, which can cause them to swell, stroke, jaundice, or yellowing of the skin, eyes, and mouth and priapism, dizziness, fatigue, low oxygen in the body, or malaise and urinary inability to make concentrated or dilute urine or blood in urine.

 

The pathology of Sickle Cell Disease often reveals that when there is insufficient oxygen in the vascular system, sickle hemoglobin becomes considerably more insoluble, increasing the polymer formation in the blood and its overall viscosity. This leads to the formation of tactoids, which are a gel-like form of hemoglobin that exists in equilibrium with its ordinary soluble state. Sickle cell disease is an inherited condition that follows an autosomal recessive pattern. When both parents carry a single gene mutation, known as the sickle cell trait, there is a 25% chance that the disease will develop in their offspring, a 25% chance that the child will be unaffected and a 50% that they will possess the genetic mutation as an asymptomatic carrier.

 

The diagnosis of a patient with SCD is done with a simple blood test and is part of routine newborn screening. In adults, a blood sample is taken from a vein in the arm. Assessing stroke risk the test uses sound waves to measure blood flow to the brain this painless test can be used in children as young as 2 years old. Tests to detect sickle cell genes before birth SCD can be diagnosed in an unborn baby by sampling some of the amniotic fluid surrounding the baby in the womb. The treatment of patients with Sickle Cell Disease can include lifestyle behaviors as well as medical screening and interventions to prevent SCD complications. Specific treatments to prevent SCD complications that can be used include the FDA approved drugs Hydroxyurea that may help people with SCD ages 2 years and older, L-glutamine that may help people with SCD ages 5 years and older, Voxelotor that may help people with SCD ages 4 years and older and Crizanlizumab that may help people with SCD ages 16 years and older. 

 

Several other treatments and therapies for SCD have recently been developed that are still undergoing clinical trials and thus have not yet been approved by the FDA.

Report Highlights

• In May 2024, BioLineRx Ltd announced a multi-center Phase I clinical trial sponsored by St. Jude Children's Research Hospital, Inc. to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with sickle cell disease (SCD).  
• In April 2024, Health Canada had granted priority review to the application of the gene-editing therapy exagamglogene autotemcel (exa-cel) for patients ages 12 and older with sickle cell disease (SCD) having recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
• In January 2024, GlycoMimetics reported positive initial data from a Phase Ia clinical trial of its E-selectin antagonist, GMI-1687, to potentially treat sickle cell disease (SCD).
• In December 2023, BioLineRx Ltd. announced that the first patient has been dosed in the Phase I clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD).
• In December 2023, the US Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.
• In August 2023, Editas Medicine expanded its partnership with Azzur Cleanrooms on Demand (COD) for the manufacturing of EDIT-301, an experimental cell-based gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia.
• In July 2023, Scribe Therapeutics expanded its collaboration with Sanofi to advance the development of a new wave of in vivo treatments for sickle cell disease (SCD) and other disorders.

Sickle Cell Disease: Company and Product Profiles (Marketed Therapies)

1. Company Overview: Vertex Pharmaceuticals

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

 

2. Product Description: CASGEVY

CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients.

 

3. Company Overview: Emmaus Medical, Inc

Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease. The company is engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. The initial efforts have focused on treatments for Sickle Cell Disease, a genetic disorder. Emmaus's lead commercial product is Endari®, an oral pharmaceutical grade L-glutamine treatment indicated to reduce acute complications of sickle cell disease in adult and pediatric patients five years of age and older.

 

Product Description: ENDARI

ENDARI [L-glutamine oral powder] is indicated to reduce the acute complications of sickle cell disease in adults and children 5 years and older. ENDARI is an amino acid indicated to reduce the acute complication of sickle cell disease in adult. The mechanism of action of the amino acid L-glutamine in treating sickle cell disease (SCD) 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. The pyridine nucleotides, NAD+ and its reduced form NADH, play roles in regulating and preventing oxidative damage in RBCs. L-glutamine may improve the NAD redox potential in sickle RBCs through increasing the availability of reduced glutathione.

Sickle Cell Disease: Company and Product Profiles (Pipeline Therapies)

Company Overview: Pfizer

Pfizer Inc. is a global biopharmaceutical company with a rich history dating back to 1849 when it was founded by Charles Pfizer and Charles Erhart in Brooklyn, New York. The company is engaged in the discovery, development, manufacture, and sale of medicines and vaccines across various therapeutic areas such as vaccines, oncology, internal medicine, hospital, inflammation and immunology, and rare diseases. Pfizer's dedication to global health is evident through its extensive portfolio of medicines and vaccines that aim to enhance wellness, prevention, treatments, and cures for various diseases. The company collaborates with healthcare providers, governments, and communities worldwide to expand access to reliable and affordable healthcare services.

 

1. Product Description: Inclacumab

Inclacumab is a fully human IgG4 anti-P-selectin monoclonal antibody, is being developed for the reduction of vaso-occlusive crises (VOCs) in patients with SCD. P-selectin is a protein that mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab has the potential to be a best-in-class option for reducing VOCs in people with SCD, with the potential for quarterly, rather than monthly dosing. The drug was being developed by the Global Blood Therapeutics (GBT), which is acquired by the Pfizer. GBT has exclusive worldwide rights to inclacumab as part of the company’s licensing agreement with Roche. The drug is currently in Phase III stage of development for the treatment of patients with Sickle Cell Disease.

 

2. Company Overview: Editas Medicine

Editas Medicine is a clinical-stage genome editing company focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines.

 

Product Description: Renizgamglogene autogedtemcel

Reni-cel, formerly known as EDIT-301, is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Reni-cel consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease. Red blood cells derived from reni-cel CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT. The drug has recently completed Phase III clinical trial for the treatment of patients with Sickle Cell Disease.

 

3. Company Overview: Roche

 Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives. The combined strengths of pharmaceuticals and diagnostics, as well as growing capabilities in the area of data-driven medical insights help Roche deliver truly personalized healthcare.

 

Product Description: RG 6107

SKY59 is a recycling antibody® discovered by Chugai that inhibits the C5 complement component. The onset of a number of diseases is reported to be caused by complement activation. SKY59 is expected to inhibit cleavage of C5 to C5a and C5b, thus suppressing complement activation and improving disease conditions. In PNH, SKY59 may have a suppressive effect on hemolysis by preventing the destruction of red blood cells. Application of multiple Chugai proprietary antibody engineering technologies resulted in a prolonged half-life, and the antibody is being developed as a subcutaneous self-injection. The drug is currently in Phase II stage of development for the treatment of patients with Sickle Cell Disease.

 

4. Company Overview: Beam Therapeutics Inc

Beam Therapeutics is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.

 

Product Description: BEAM-101

BEAM-101 is an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). The one-time therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promotor regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. HbF is the predominant hemoglobin variant during development and early life. The safety and efficacy of BEAM-101 is being evaluated in the ongoing BEACON Phase I/II study, an open-label, single-arm, multicenter trial in adult patients with SCD.

 

5. Company Overview: BRL Medicine

VYNE’s mission is to improve the lives of patients by developing proprietary, innovative, and differentiated therapies for the treatment of immuno-inflammatory conditions. The Company’s unique and proprietary pipeline includes access to a library of bromodomain & extra-terminal (BET) domain inhibitors licensed from In4Derm Limited. The BET inhibitor platform includes lead programs and access to a library of (BET) domain inhibitors for the potential treatment of immuno-inflammatory conditions. 

 

Product Description: BRL 101

In August 2022, the clinical trial application (IND) of BRL Medicine's BRL-101 was officially approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, entering the registration clinical stage. Its primary indication is transfusion-dependent beta-thalassemia, developed as a gene therapy product based on the independently developed hematopoietic stem cell platform (ModiHSC®) by BRL Medicine. Currently, BRL Medicine's gene therapy, BRL-101, has successfully enabled 15 thalassemia patients worldwide to become independent of blood transfusions. The results of the IIT and IND Phase I clinical studies of BRL-101 were announced at this meeting. The study, conducted in China, evaluated ""gamma globin reactivated autologous hematopoietic stem cell transplantation for the treatment of β-thalassemia major (Thalassemia) safety and effectiveness."" The clinical results indicated a significant increase in overall Hb and HbF levels after all patients received gene-edited HSC transplantation. Throughout the treatment process, most adverse events were consistent with known adverse events for hematopoietic stem cell mobilization/apheresis, busulfan myeloablative conditioning, and autologous hematopoietic stem cell transplantation. All adverse events could be resolved with medical intervention, and the vast majority of adverse events were resolved. There were no cases of GVHD, subject withdrawal from the study, or deaths due to adverse events. The drug is currently in preclinical stage of development for the treatment of patients with Sickle Cell Disease.

Explore the evolving Sickle Cell Disease Market trends, key players, and future outlook.

Sickle Cell Disease Analytical Perspective by DelveInsight

• In-depth Commercial Assessment: Sickle Cell Disease 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 Competitive Landscape 

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

• Sickle Cell Disease Report Assessment
• Company Analysis
• 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 and approved drugs?