ASH 2025 Spotlight: Transformative Sickle Cell Disease Clinical Developments
Akshat Jain, Hematology Pediatric Workforce Task Force at American Society of Hematology, shared on LinkedIn:
”A day filled with remarkable science in clinical development for Sickle Cell Disease at the American Society of Hematology 2025 national meeting.
Using novel new ways to positively affect the Erythron, three new approaches were discussed :
1. Transcriptome guided novel target by affecting NEDDylation through DCN1 pathway, thereby augmenting fetal hemoglobin rise, predicted to raise fetal hemoglobin close to gene therapy ranges by just an oral drug delivery platform.
2. Knockdown of G-CSF in Sickle Cell Disease, hoping it will impact Netosis and vaso occlusion. Thromboinflammation front and center. Very complimentary to our work being done by the Sickle Cell Hemostasis Taskforce.
3. Acute chest syndrome: acute treatment by reduction of NETs
Additionally proud to have shared our clinical work on standardizing point of care testing with near 100% concordance with HPLC for all hemoglobinopathies in the USA for the first time.”
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