Beta-thalassemia is an inherited hemoglobin disorder caused by reduced or absent beta-globin chain production, resulting in chronic anemia of variable severity.
The FDA has approved Agios Pharmaceuticals' mitapivat (AQVESME) as the first oral treatment for anemia in thalassemia, reducing transfusion needs by 40-50%. Meanwhile, a clinical trial found a three-times-weekly dosing of vadadustat (Vafseo) is safe for kidney dialysis patients, potentially improving convenience.
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.
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.
The FDA has issued draft guidance that may speed approval pathways for rare disease therapies using genome editing technologies, potentially benefiting CRISPR Therapeutics' pipeline of six clinical-stage candidates.
Switzerland-based CRISPR Therapeutics stands to benefit from increased Casgevy sales following partner Vertex Pharmaceuticals' fourth-quarter results, with five additional gene-editing therapies in clinical trials targeting larger patient populations.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07681440 |
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia |
TERMINATED | |
| NCT07599176 |
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors |
RECRUITING | PHASE1/PHASE2 |
| NCT06831799 |
ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 |
COMPLETED | |
| NCT06772766 |
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia |
RECRUITING | PHASE1 |
| NCT06717932 |
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection |
ENROLLING_BY_INVITATION | |
| NCT06685536 |
A Long-term Follow-up Study in Participants Who Received CS-101 |
ENROLLING_BY_INVITATION | |
| NCT06647979 |
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies |
RECRUITING | PHASE1 |
| NCT06568926 |
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy |
RECRUITING | NA |
| NCT06539169 |
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases |
RECRUITING | |
| NCT06512298 |
Plasticizer Exposure and Its Consequences on Health |
NOT_YET_RECRUITING |
