News

New data presented by AyrmidPharma at #ASH2025 indicates that motixafortide (APHEXDA®) can robustly mobilize the hematopoietic stem cells (HSCs) required for gene therapies in sickle cell disease (SCD). The Phase …

The U.S. FDA has approved Agios Pharmaceuticals‘ AQVESME™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia. This approval, based on the positive Phase 3 ENERGIZE and …

New Phase 1b data from Fulcrum Therapeutics suggest that higher doses of the investigational oral therapy pociredir may offer greater benefits for adults with sickle cell disease (SCD). In the …

New clinical data from Ayrmid highlight a promising advance in stem cell mobilization for people with SCD pursuing gene therapy. In a first-in-human trial, the CXCR4 inhibitor motixafortide, alone and …

New laboratory research from Vascarta Inc. highlights a novel approach to addressing SCD complications. The company’s investigational skin gel, VAS‑101, which contains the turmeric-derived compound curcumin, partially restored mitochondrial function …

Novartis announced it will present data from over 70 abstracts at the upcoming American Society of Hematology (ASH) Annual Meeting and San Antonio Breast Cancer Symposium (SABCS). Key highlights include …

Agios Pharmaceuticals has announced positive top-line results from its Phase 3 RISE UP trial, demonstrating that its oral therapy, mitapivat, significantly increased hemoglobin levels in patients with sickle cell disease …

Disc Medicine has initiated a Phase 1b clinical trial for DISC-3405, an investigational therapy designed to address iron overload in patients with sickle cell disease (SCD). The trial will evaluate …

We’re now just days away from the Mid-Atlantic Capital Conference, and the excitement at OxyDial continues to build as we prepare to take the stage on October 30th at the …