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This company develops cell and gene therapies specifically targeting rare pediatric diseases. They utilize proprietary CRISPR 3.0 technology for precise gene editing to correct genetic mutations in patient cells. Their pipeline includes ex vivo cell therapies that have received Orphan Drug Designation from both the EMA and FDA for severe genetic disorders.
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Updated 4 months ago
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Product
Features
- This company develops cell and gene therapies specifically targeting rare pediatric diseases. They utilize proprietary CRISPR 3.0 technology for precise gene editing to correct genetic mutations in patient cells. Their pipeline includes ex vivo cell therapies that have received Orphan Drug Designation from both the EMA and FDA for severe genetic disorders.