Also known as: sickle cell
Sickle cell disease is a group of inherited red blood cell disorders caused by abnormal hemoglobin. Red blood cells become rigid and sickle-shaped, leading to vaso-occlusion, pain crises, hemolysis, and organ damage.
New research shows that many people with disease-causing gene mutations never develop illness, challenging traditional assumptions. Studies of 'modifier genes' and genetic outliers are revealing how some individuals stay healthy, potentially leading to new treatments for genetic diseases.
Crispr Therapeutics AG trades at $47.78 with a $4.78B market cap, while Moderna trades at $61.82 with a $25.06B market cap, about 5.2 times larger. Both face losses, but CRISPR's Casgevy approval and Moderna's pipeline advances shape divergent analyst outlooks.
Data presented at EHA show that sickle cell disease patients have high interest in gene therapy, with 71% eager to learn more, despite limited awareness and concerns about safety and cost. Early clinical data from approved CRISPR-based therapies demonstrate significant reductions in vaso-occlusive crises and hospital admissions.
Vertex Pharmaceuticals dominates cystic fibrosis therapies with TRIKAFTA/KAFTRIO generating $2.57 billion in Q4 2025 and ALYFTREK ramping to $380.1 million. The company posted strong free cash flow of $3.19 billion in 2025 and is expanding into pain, kidney disease, and gene-editing therapies.
Canaccord Genuity initiated Beam Therapeutics with a buy rating and $74 price target, highlighting near-term catalysts. Beam reached an agreement with the FDA on an accelerated approval pathway for its BEAM-302 AATD therapy, which showed early clinical proof-of-concept for in vivo base editing. The company plans a BLA for risto-cel and a pivotal plan for BEAM-302 in early 2026.
CRISPR Therapeutics said CASGEVY launch momentum is building and multiple pipeline assets could generate data in the next 12 to 18 months. The company also highlighted zugo-cel progress in oncology and autoimmune disease.
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
Three advances in TB diagnostics: a portable $300 device delivering results in 30 minutes, a Quick DNA kit detecting drug resistance in 5 minutes, and an ultrasensitive test finding TB DNA in 12-16% of hospitalized US patients.
Vertex completed the rolling BLA submission for povetacicept in IgA nephropathy after positive Phase 3 RAINIER interim data. The company plans to use a Priority Review Voucher to accelerate FDA review.
More than 30 approved cell and gene therapies carry prices from nearly $1 million to over $4 million, creating fiscal pressure for Medicaid. A federal access model launched in 2024 aims to centralize outcomes-based contracting.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07776444 |
Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography |
RECRUITING | NA |
| NCT07753681 |
Domestication and Implementation of the PEN-Plus Clinical Model in the Zambian Health System |
RECRUITING | |
| NCT07721155 |
Objective Neurocognitive Assessment of Young Children With Sickle Cell Disease by Eye-Tracking |
COMPLETED | |
| NCT07719972 |
Universal Newborn Screening For Sickle Cell Disease In Mozambique |
NOT_YET_RECRUITING | NA |
| NCT07708714 |
Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study |
RECRUITING | PHASE1/PHASE2 |
| NCT07708350 |
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease |
NOT_YET_RECRUITING | PHASE1 |
| NCT07674277 |
Virtual Reality for SCD VOC |
NOT_YET_RECRUITING | NA |
| NCT07656415 |
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) |
RECRUITING | PHASE3 |
| NCT07648043 |
Feasibility and Acceptability of Amani |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07640815 |
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease |
TEMPORARILY_NOT_AVAILABLE |
