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BAK1 stimRNA
- Development stage
- Preclinical
- Lead developer
- Thomas Jefferson University
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
BAK1 stimRNA is a megakaryocyte- and platelet-selective naked modified small interfering RNA (siRNA) designed to suppress the expression of the pro-apoptotic protein BCL-2 homologous antagonist/killer 1 (BAK1). Developed by researchers at Thomas Jefferson University, this translation-inhibiting RNA therapy aims to mitigate platelet storage lesion (PSL) and enhance the lifespan and function of transfused platelets. By downregulating BAK1, the therapy prevents platelet apoptosis, phosphatidylserine (PS) exposure, and premature platelet clearance, thereby improving platelet preservation ex vivo and increasing circulating platelet counts in vivo.
- Other names
- BAK1 platelet-selective naked modified siRNABAK-1 platelet-selective naked modified siRNABAK 1 platelet-selective naked modified siRNABAK1 stimRNAsBAK-1 stimRNAsBAK 1 stimRNAs
02
Targets
Bcl-2 homologous antagonist/killer 1 (BAK1) mRNABAK
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