Sickle cell disease affects about 100,000 people in the United States, causing red blood cells to form a crescent shape that blocks blood flow and triggers pain crises. Until recently, treatment focused on managing symptoms and complications. Two gene therapies approved in 2023 can now cure the disease in eligible patients.
What's actually going on in research
Active trials are testing gene therapies that either edit the sickle mutation directly or reactivate fetal hemoglobin, gene-addition approaches, and CRISPR-based editing. Researchers are also studying new drugs to prevent pain crises, reduce organ damage, and make gene therapy safer and more accessible. Trials are exploring ways to avoid chemotherapy conditioning and reduce treatment time.
Gene therapy expansion
Two gene therapies are now FDA-approved: exagamglogene autotemcel and lovotibeglogene autotemcel. Ongoing trials aim to make these treatments faster, safer, and available to more patients.
CRISPR gene editing
Direct editing of the sickle mutation in stem cells shows promise in early trials. This approach could potentially offer more precise correction than current gene-addition methods.
Pain crisis prevention
New drugs targeting inflammation and blood flow are being tested to reduce the frequency and severity of pain crises. Crizanlizumab and voxelotor represent recent additions, with more in development.
What to know before you search
Eligibility typically depends on disease severity, frequency of pain crises or complications, age, and whether prior treatments like hydroxyurea have been tried.
What types of trials are currently open
- Gene therapy trials — Testing treatments that modify or replace stem cells to produce healthy hemoglobin. These require stem cell collection, lab modification, chemotherapy conditioning, and infusion of edited cells.
- Gene editing trials — Studies using CRISPR or similar tools to directly correct the sickle mutation in a patient's own stem cells.
- Pain crisis trials — Testing drugs that aim to prevent or reduce pain crises by preventing red cell sickling or reducing inflammation.
- Organ protection trials — Studies of treatments to prevent or slow kidney damage, stroke, lung complications, and other organ problems caused by sickle cell disease.
- Long-term follow-up studies — Tracking patients who received gene therapy or other new treatments to monitor durability and long-term safety.
Recently added Sickle Cell Disease trials
Participate in a new digital screening and care coordination system
A cluster randomized trial (CRT) of the novel sickle cell disease (SCD), M-health based SCD Information Management and Communication system SIMCS vs routine new born screening strategy in health care centres in Uganda to evaluate impact on access screening, coordination of care and clinical outcomes
Receive a stem cell transplant from a matched donor
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.
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