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CD47 morpholino oligonucleotide
- Development stage
- Preclinical
- Lead developer
- San Rocco Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous, Intraperitoneal, Intravenous
01
Overview
CD47 morpholino oligonucleotide is a preclinical-stage antisense therapy being developed by Errant Gene Therapeutics (also known as San Rocco Therapeutics) in collaboration with Dr. Jeff Isenberg and Research Corporation Technologies (RCT). The drug utilizes morpholino oligonucleotide technology to modulate the expression of CD47, a cell surface protein that serves as an immune checkpoint and a "don't eat me" signal. By targeting CD47, the therapy aims to treat Beta-Thalassemia, potentially by preventing the premature clearance of red blood cells and improving erythropoiesis. This approach represents a new generation of oligonucleotide-based treatments for hemoglobinopathies.
- Other names
- CD47 morpholinoCD-47 morpholinoCD 47 morpholinoCD47-morpholino oligonucleotideCD-47-morpholino oligonucleotideCD 47-morpholino oligonucleotide
02
Targets
CD47 (Cluster of Differentiation 47)
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