Top Companies in the Developing Market and Market Growth
Market Growth
The worldwide gene therapy for sickle cell disease market is experiencing significant expansion, with projections indicating a revenue increase reaching several hundred million dollars by the end of the forecast period, spanning 2025 to 2034. This growth is driven by emerging trends and strong demand across key sectors.
Trends in the Gene Therapy for Sickle Cell Disease Market
A surge in the development of more effective and permanent treatments, and expanded patient awareness, is widely impacting the overall market expansion.
- In July 2025, Red blood cell health company Functional Fluidics entered into a collaboration with Safi Biotherapeutics to validate the health and quality of Safi’s stem-cell-derived, manufactured human red blood cell (mRBC) products for SCD.
- In March 2025, Zynext Ventures invested in Illexcor Therapeutics to advance the clinical development of ILX002, its lead, a new oral candidate therapy for sickle cell disease (SCD).
- In December 2024, Tessera received $50M from the Gates Foundation to advance in vivo sickle cell gene therapy.
The Market Value Chain Analysis
R&D
The gene therapy for sickle cell disease market includes the identification of a gene target, the collection of a patient's blood-forming stem cells, and then the reinfusion of the modified cells into the patient after conditioning to make space in the bone marrow.
Key Players: Vertex Pharmaceuticals, CRISPR Therapeutics, Bluebird Bio, etc.
Clinical Trials and Regulatory Approvals
In this stage, subjects are recruited, and consent is obtained for a complex process in which their stem cells are going to be collected and modified for the detection of required safety, efficacy, and adverse effects. And, further, they will acquire the FDA's Biologics License Application (BLA) for approval.
Key Players: David Williams, Washington University School of Medicine, Essen Biotech, Memorial Sloan Kettering Cancer Center, etc.
Patient Support and Services
This can be done through pre-treatment consultation with physicians, long-term clinical follow-up (often 2-3 years in a hospital and ongoing outpatient visits), and the probability of community-based patient registries for tracking long-term results.
Key Players: Gates Foundation, Novartis, Precision BioSciences, etc.
Latest Announcements by Industry Leaders
- In August 2025, Vertex Pharmaceuticals and Orna Therapeutics announced a three-year collaboration to design next-generation gene therapies for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Orna’s CEO, Amit Munshi, said that this collaboration will support leveraging their proprietary technologies to achieve unprecedented delivery to haematopoietic stem cells.
- In April 2025, Xcellbio announced a commercial license and entered into a supply agreement for the use of Xcellbio’s proprietary cell manufacturing technology, AVATAR, to support the commercialization of LYFGENIA gene therapy for the treatment of sickle cell disease. Brian Feth, CEO of Xcellbio, replied that this will expand their business strategy of assisting new modalities to bring transformative treatment options to patients.
What are the Recent Developments in the Respective Market?
- In August 2025, Beam Therapeutics Inc., a biotechnology company developing precision genetic medicines through base editing, launched its Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD).
- In July 2025, the Centers for Medicare & Medicaid Services (CMS) unveiled its Cell and Gene Therapy (CGT) Access Model, an innovative approach to delivering cutting-edge treatments for people on Medicaid living with sickle cell disease.
- In April 2025, the Department of Health, Abu Dhabi (DoH), introduced CASGEVY, a CRISPR/Cas9 gene-editing therapy focused on the treatment of sickle cell disease and transfusion-dependent beta-thalassemia.
Become a valued research partner with us, please feel free to contact us at sales@towardshealthcare.com
