From anemia and blood clots to serious diseases of the blood, such as sickle cell disease and leukemia, millions of people are affected by blood disorders each year. There is a great need for solutions to better diagnose and treat blood disorders.

In recent years, precision-medicine approaches, advances in the study of genetics and gene therapies have provided hope to patients living with the devastating effects of blood disorders.

2026 Blood Disorder Statistics

• Approximately 900,000 people develop blood clots annually in the U.S.¹ Approximately 100,000 people die each year from blood clots.¹
• The prevalence of anemia in people 2 years of age and older is 9.3%, with higher prevalence in females (13.0%) than in males (5.5%).²
• In hemophilia, nearly 100% of patients in high-income countries receive a diagnosis; in lower-income countries, the diagnosis rate is as low as 12%.³
• Approximately 7.74 million people live with sickle-cell disease globally.⁴
• The most common cancer in children and people younger than 20 is Leukemia.⁵
• Leukemia is most prevalent in people over 60.⁵

A New Therapy for a Rare Cause of Anemia

Warm autoimmune hemolytic anemia (wAIHA) is a rare blood disorder where the immune system attacks and destroys healthy red blood cells. Patients living with this condition may experience severe fatigue, weakness, pale skin and jaundice, dizziness, palpitations and difficulty breathing.⁶ In 2025, the FDA approved the first therapeutic for the treatment of this life-threatening condition. Johnson&Johnson’s IMAAVY®️ (nipocalimab-aahu) is an immunoselective FcRn blocker that reduces pathogenic immunoglobulin G (IgG) autoantibodies and preserves B-cell function. In a randomized, placebo-controlled trial, three times as many patients receiving IMAAVY achieved durable Hgb levels versus placebo, and use of IMAAVY was associated with less fatigue. Overall, this new treatment is a life-changing solution for people struggling with the effects of this rare, debilitating condition.⁷

Powering The Prediction of Venous Thromboembolism (VTE)

Venous thromboembolism (VTE) occurs when a blood clot forms in a deep vein, typically in the the leg or thigh, and includes conditions like deep venous thrombosis (DVT) and pulmonary embolism (PE). Though these are common, life-threatening cardiovascular diseases, their causes are not completely understood. New research into the biological mechanisms of VTE may help identify people at risk and spur new preventive approaches and treatments. Using large-scale, high-throughput aptamer-based proteomics, a recent study identified novel plasma proteins reflecting the biological processes of VTE pathophysiology, including extracellular matrix regulation, immunity, immune–vascular endothelium interactions, and vascular senescence. These results could provide potential targets that may improve methods for VTE prevention, risk prediction, and the development of new treatments.⁸

Promising New Research in Hemophilia 

In recent years, new genetic discoveries and the use of gene therapy have led to new treatments for hemophilia A.

An investigational antibody therapy, Denecimig (Mim8), by Novo Nordisk, showed low bleeding rates in individuals with hemophilia A in an ongoing, open-label extension study. The therapy is designed to prevent or reduce bleeding, while supporting the blood’s ability to clot.⁹

During a prospective study of patients with thrombosis, stroke and other blood clotting disorders, researchers at CU Anschutz identified a rare, naturally occurring gain-of-function mutation in the critical blood-clotting protein FVIII. The newly discovered genetic variant, FVIII Aurora, improves FVIII’s stability and activity, and may offer a new therapeutic target for hemophilia A.¹⁰

The FDA recently granted fast track status to a one-time stem cell therapy for hemophilia A. In a Phase 1 trial, this promising therapy led to increased levels of FVIII, eliminating a need for preventive treatment.¹¹

Gene Therapy for Sickle Cell Disease

The FDA approved the first gene therapy for children 2 years and older with sickle cell disease.¹²  Casgevy (exagamglogene autotemcel) utilizes CRISPR/Cas9, a type of genome editing technology, to modify a patient’s blood stem cells. The modified blood stem cells are transplanted back into the patient’s body where they increase production of fetal hemoglobin and prevent red blood cells from sickling.¹³ Because Casgevy is considered highly invasive and costly, more accessible solutions are needed.¹⁴

Advances in Blood Cancer Treatment

According to recent analyses, treatment advances in blood cancers have saved more than 25 million years of life in the U.S.¹⁵  New cell therapies and personalized-medicine approaches are improving the quality of life of people living with certain blood cancers.

In Stanford Cancer Institute’s multicenter, phase 3 trial, Precision-T, researchers explored Orca-T (TEGRZI), a cell therapy that utilizes regulatory T cells to prevent immune overactivation, which occurs when a treatment attacks healthy tissue in addition to the aggressive cancer. The study demonstrated improved survival rates and decreased side effects. Orca-T recently received FDA approval and is the first regulatory T cell-based therapy approved for clinical use.

Experts at The Institute of Cancer Research in London, exploring a personalized-medicine approach to treating patients with high-risk multiple myeloma, provided recently diagnosed people with custom treatments, combining a stem cell transplant and a variety of drugs. After six years, 70% of patients who participated in the trial were still alive, compared to 40% receiving standard therapy.¹⁶

New research and treatments for blood disorders have accelerated personalized care for the millions of patients living with the effects of these often serious conditions. From improved diagnostics to precision gene therapies, new technology and approaches to treatment are improving quality of life and delivering life-saving care faster.

Sources:

1. https://www.hematology.org/education/patients/blood-disorders/fact-sheets
2. https://www.cdc.gov/nchs/products/databriefs/db519.htm
3. https://pmc.ncbi.nlm.nih.gov/articles/PMC10772874/
4. https://www.who.int/news-room/fact-sheets/detail/sickle-cell-disease
5. https://bloodcancerunited.org/resources/blog/5-essential-facts-you-should-know-about-leukemia
6. https://rarediseases.org/rare-diseases/warm-autoimmune-hemolytic-anemia/
7. https://www.jnj.com/media-center/press-releases/fda-approves-imaavy-nipocalimab-aahu-as-first-ever-treatment-for-warm-autoimmune-hemolytic-anemia-waiha-representing-a-landmark-advancement-for-patients
8. https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.125.074493
9. https://hemophilianewstoday.com/news/experimental-treatment-maintains-low-bleed-rates-hemophilia-a/
10. https://www.childrenscolorado.org/advances-answers/recent-articles/genetic-hemophilia-a-treatment/
11. https://hemophilianewstoday.com/news/fda-grants-fast-track-rare-pediatric-disease-status-stem-cell-therapy-hemophilia-a/
12. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-young-children-sickle-cell-disease
13. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
14. https://frontlinegenomics.com/sickle-cell-disease-a-2024-update/
15. https://www.hematology.org/newsroom/press-releases/2026/investment-in-blood-cancer-research-has-saved-nearly-26-million-years-of-life
16. https://www.independent.co.uk/news/uk/home-news/blood-cancer-ken-theobold-test-myeloma-trial-b3046428.html