Sickle Cell Disease Treatment Pipeline: Detailed 2025 Insights

DelveInsight's "Sickle Cell Disease Pipeline Insights 2025" analysis delivers extensive perspectives on 55+ organizations and 60+ therapeutic candidates in the Sickle Cell Disease Competitive environment. It encompasses Sickle Cell Disease Pipeline Treatment evaluation by product classification, developmental phase, administration route, and molecular category. The analysis further spotlights the dormant pipeline assets in this therapeutic domain.

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Essential Highlights from the Sickle Cell Disease Pipeline Analysis

On 10 October 2025, Novo Nordisk A/S initiated an investigation designed to validate whether etavopivat demonstrates efficacy in diminishing the frequency of Vaso-occlusive crisis VOCs (sickle cell pain episodes) triggered by obstructions in blood vessels among adults and adolescents diagnosed with sickle cell disease. The investigation will additionally assess how effectively etavopivat can minimize organ damage, enhance exercise capacity and diminish fatigue in individuals with sickle cell disease. Participants will receive either etavopivat or placebo. Treatment assignment for participants will be determined randomly.

On 02 October 2025, Pfizer launched an investigation representing an open-label study to assess the safety of extended administration of inclacumab in subjects with sickle cell disease (SCD). Subjects in this investigation will have successfully completed a previous study of inclacumab.

DelveInsight's Sickle Cell Disease pipeline analysis reveals a dynamic landscape with 55+ active organizations engaged in advancing 60+ pipeline candidates for Sickle Cell Disease management.

Prominent Sickle Cell Disease pharmaceutical developers include Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc, GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, Base Therapeutics among others.

Promising Sickle Cell Disease Pipeline Treatment Assets include Siplizumab, Daratumumab, Alemtuzumab, Sirolimus, ITU512, BEAM-101, Crizanlizumab, Etavopivat, Epeleuton, Voxelotor among others.

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The Sickle Cell Disease Pipeline Analysis offers disease background, pipeline landscape and therapeutic evaluation of the principal pipeline candidates in this field. The Sickle Cell Disease Pipeline Analysis additionally emphasizes the unaddressed medical needs concerning Sickle Cell Disease.

Understanding Sickle Cell Disease

Sickle Cell Disease represents the designation for a collection of inherited conditions affecting red blood cells with the most severe variant termed Sickle cell anemia. Sickle cells undergo rapid destruction in the bodies of individuals with the condition, triggering anemia which explains why it is frequently referred to as sickle cell anemia. The sickle cells additionally obstruct blood flow through vessels, producing lung tissue injury that triggers acute chest syndrome, pain episodes, stroke, priapism and injury to the spleen, kidneys and liver.

Sickle Cell Disease Approved Treatments

Vertex Pharmaceuticals: CASGEVY Vertex operates as a global biotechnology enterprise investing in scientific innovation to generate transformative medicines for individuals with serious conditions. The organization has regulatory-approved medicines addressing the underlying causes of multiple chronic, life-shortening genetic disorders including cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia and continues to progress clinical and research programs in these conditions. Vertex additionally maintains a robust clinical pipeline of investigational treatments across a spectrum of modalities in other serious conditions where it possesses deep understanding of causal human biology, encompassing APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

CASGEVY™ represents a non-viral, ex vivo CRISPR/Cas9 gene-edited cellular therapy for eligible individuals with SCD or TDT, wherein a patient's own hematopoietic stem and progenitor cells undergo editing at the erythroid specific enhancer region of the BCL11A gene via a precise double-strand break. This modification produces elevated concentrations of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF represents the variant of the oxygen-carrying hemoglobin naturally present throughout fetal development, which subsequently transitions to the adult variant of hemoglobin following birth. CASGEVY has demonstrated capability to diminish or eliminate VOCs for individuals with SCD and transfusion requirements for individuals with TDT. CASGEVY has received approval for certain indications in multiple jurisdictions for eligible individuals.

Emmaus Medical Inc.: ENDARI Emmaus Life Sciences, Inc. operates as a commercial-stage biopharmaceutical enterprise and pioneer in the management of sickle cell disease. The organization engages in the discovery, development and commercialization of innovative treatments and therapies predominantly for rare and orphan conditions. The initial initiatives have concentrated on treatments for Sickle Cell Disease, a genetic disorder. Emmaus's principal commercial product is Endari®, an oral pharmaceutical grade L-glutamine treatment indicated to diminish acute complications of sickle cell disease in adult and pediatric individuals five years of age and above.

ENDARI [L-glutamine oral powder] is indicated to diminish the acute complications of sickle cell disease in adults and children 5 years and above. ENDARI represents an amino acid indicated to diminish the acute complication of sickle cell disease in adults. The mechanism of action of the amino acid L-glutamine in managing sickle cell disease (SCD) is not completely characterized. Oxidative stress mechanisms are implicated in the pathophysiology of SCD. Sickle red blood cells (RBCs) demonstrate greater susceptibility to oxidative damage compared to normal RBCs, which may contribute to the chronic hemolysis and vaso-occlusive events associated with SCD. The pyridine nucleotides, NAD+ and its reduced variant NADH, fulfill roles in regulating and preventing oxidative damage in RBCs. L-glutamine may enhance the NAD redox potential in sickle RBCs through increasing the availability of reduced glutathione.

The Sickle Cell Disease Pipeline Analysis Delivers Perspectives on

The analysis offers comprehensive insights regarding organizations advancing treatments for Sickle Cell Disease management with aggregate therapeutic candidates developed by individual companies for this indication.

It evaluates various therapeutic assets categorized into initial-stage, intermediate-stage, and advanced-stage development for Sickle Cell Disease Treatment.

Sickle Cell Disease pharmaceutical developers engaged in targeted therapeutic advancement with corresponding active and inactive (dormant or terminated) programs.

Sickle Cell Disease therapeutics under investigation classified by development phase, administration route, target receptor, monotherapy or combination approaches, distinct mechanisms of action, and molecular classification.

Comprehensive examination of partnerships (inter-company collaborations and company-academic institution partnerships), licensing agreements and funding arrangements for future progression of the Sickle Cell Disease market.

Investigational Sickle Cell Disease Therapeutics Across Various Clinical Development Phases Comprise:

• RL 101: BRL Medicine

• ORY-300:1 Oryzon Genomics

• GSK 4172239D: GlaxoSmithKline

• AG-946: Agios Pharmaceuticals

• BEAM-101: Beam Therapeutics Inc.

• Nula-cel: Graphite Bio

• RG 6107: Roche

• Isoquercetin: Quercis Pharma

• Renizgamglogene autogedtemcel: Editas Medicin

• Inclacumab: Pfizer

• L-glutamine: Emmaus Medical, Inc

• CASGEVY: Vertex Pharmaceuticals

• ESCAPE: Beam Therapeutic

• IHP-102: IHP Therapeutics

• HBI-002: Hillhurst Biopharmaceuticals

• BEAM101: Beam Therapeutics

• EPI01: Novo Nordisk

• VIT-2763: CSL Vifor

• Inclacumab: Pfizer

• L-glutamine: Emmaus Medical

• Oxbryta: Pfizer

• Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals

• Mitapivat: Agios Pharmaceuticals

• Canakinumab: Novartis

• ALXN1820: Alexion Pharmaceuticals

• Crovalimab: Chugai Pharmaceutical/Roche

• EDIT 301: Editas Medicine

• BIVV003: Sangamo Therapeutics

• BEAM101: Beam Therapeutics

• Hemopexin: CSL Behring

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Sickle Cell Disease Pharmaceutical Developers

Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc, GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, Base Therapeutics, among others.

The Sickle Cell Disease Pipeline Analysis delivers therapeutic classification of pipeline candidates by Administration Route. Assets have been organized across various administration methods including:

• Intranasal

• Intrathecal

• Intravenous

• Oral

• Oral/Intravenous

• Parenteral

• Subcutaneous

• Subcutaneous/Intramuscular

• Transdermal

Sickle Cell Disease Assets have been categorized across various Molecular classifications including:

• Antisense oligonucleotide

• Gene therapy

• Hormones

• Neuropeptides

• Oligonucleotides

• Small Molecule

• Triglyceride

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Parameters of the Sickle Cell Disease Pipeline Analysis

Geographic Coverage: Global

Sickle Cell Disease Pharmaceutical Organizations: Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc, GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, Base Therapeutics among others.

Sickle Cell Disease Pipeline Treatment Assets: Siplizumab, Daratumumab, Alemtuzumab, Sirolimus, ITU512, BEAM-101, Crizanlizumab, Etavopivat, Epeleuton, Voxelotor among others.

Sickle Cell Disease Treatment Evaluation by Product Classification: Monotherapy, Combination therapy, Monotherapy/Combination therapy

Sickle Cell Disease Treatment Evaluation by Clinical Phases: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Report Contents

• Introduction

• Executive Summary

• Sickle Cell Disease (SCD): Disease Overview

• Sickle Cell Disease - Strategic Perspective: In-depth Commercial Evaluation

• Competitive Environment

• Sickle Cell Disease: Company and Product Profiles (Approved Treatments)

  • Vertex Pharmaceuticals

  • CASGEVY

• Sickle Cell Disease: Company and Product Profiles (Pipeline Treatments)

• Advanced Stage Assets (Phase III)

  • Pfizer

  • Inclacumab

  • Drug profiles in the comprehensive analysis…..

• Intermediate Stage Assets (Phase II)

  • Roche

  • RG 6107

  • Drug profiles in the comprehensive analysis…..

• Initial Stage Assets (Phase I/II)

  • Beam Therapeutics

  • BEAM-101

  • Drug profiles in the comprehensive analysis…..

• Preclinical and Discovery Phase Assets

  • BRL Medicine

  • BRL 101

  • Drug profiles in the comprehensive analysis…..

• Discontinued Assets

• Sickle Cell Disease- Unaddressed Medical Needs

• Sickle Cell Disease – Market Growth Catalysts and Obstacles

• Supplementary Materials

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