Where sickle cell disease treatment is heading

About 100,000 Americans live with sickle cell disease, an inherited blood disorder that causes painful crises, organ damage and shortened life. Hydroxyurea remains the backbone. Two gene therapies approved in 2023 can effectively cure it but are hard to access, and a new class of daily pills (pyruvate kinase activators) is nearing approval.

• 269studies open or running
• 24phase 3 trials
• 1,649papers published this year
• 40NIH-funded projects

1. Now

   Nov 2026

   Gene editing ends pain crises for mostHappened 97% crisis-free 12+ months

   Casgevy: 97% had no pain crises for a year or more; approved down to age 2 in July 2026.

   97%: free of pain crises for at least 12 months.

   • Exa-cel (Casgevy) trial

   Half-matched transplants workHappened 95.0% alive

   Transplants from half-matched relatives: 95.0% of adults alive and most cured.

   95.0%: alive after transplant.

   • Half-matched transplant trial

   New daily pills succeed in phase 3Happened 48.7% hemoglobin response

   Etavopivat raised hemoglobin in 48.7% and cut pain crises by 27%; mitapivat also raised hemoglobin.

   48.7%: hemoglobin response with etavopivat.

   • Pyruvate kinase activator trials

2. 6 months

   by May 2027

   Filings for the new pillsLikely

   Novo Nordisk planned an etavopivat filing for late 2026; Agios is pursuing a path for mitapivat.

   • Pyruvate kinase activator trials

3. 12 months

   by Nov 2027

   A second etavopivat trialPossible

   HIBISCUS 2 (510 people) finishes August 2027.

   • HIBISCUS 2

   A BTK pill for pain crisesLong shot

   Sanofi's rilzabrutinib (LIBRA, 192 people) is tested to prevent pain crises; due July 2027.

   • LIBRA

4. 3 years

   by late 2029

   Gentler gene therapyLong shot 80% fewer crises

   Reduced-intensity conditioning cut pain crises by 80% or more in all 7 early patients; larger trials are needed.

   80%: or greater reduction in severe pain crises in all 7 patients.

   • Gentler gene therapy trial

   A next-generation hemoglobin modifierLong shot

   Pfizer's osivelotor, successor to the withdrawn voxelotor, is in a phase 2/3 trial; due 2030.

   • Osivelotor trial

5. 5 years

   by late 2031

   Cures without chemotherapyLong shot

   Research aims at in-body gene editing and conditioning without chemotherapy, which would make cures far more accessible.

   • Gentler gene therapy trial
   • Exa-cel (Casgevy) trial

The research paths

Borrowed from other conditions

Open to join now

• Ages 12+

  Etavopivat

  Phase 3 · 175 sites

  Locations and eligibility →

• Adults and adolescents

  Osivelotor

  Phase 2/3 · 80 sites

  Locations and eligibility →

• Ages 10-65

  Rilzabrutinib

  Phase 3 · 63 sites

  Locations and eligibility →

• Pain crises

  Crizanlizumab

  Phase 3 · 36 sites

  Locations and eligibility →

See every recruiting Sickle cell disease trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

What just happened

Standard treatment today

• All patientsHydroxyurea; vaccines and penicillin in children; transcranial Doppler screening for stroke risk.
• Frequent pain crisesHydroxyurea, L-glutamine or crizanlizumab; transfusions for some.
• Severe diseaseGene therapy (Casgevy or Lyfgenia) or stem cell transplant from a matched or half-matched donor.
• Pain crisisFast, adequate pain relief; fluids; treatment of acute chest syndrome.

See Sickle cell disease trials recruiting now →