CAMP4 Therapeutics’ CMP-CPS-001 Receives the US FDA’s Rare Pediatric Disease Designation to Treat Urea Cycle Disorders
Shots:
- CAMP4 Therapeutics’ CMP-CPS-001 has received rare pediatric disease designation from the US FDA to treat urea cycle disorders (UCDs)
- CMP-CPS-001 (SC) is being assessed under the P-I study for the safety, tolerability, pharmacokinetics & pharmacodynamics of its single & multiple ascending doses among healthy adults in Australia
- CMP-CPS-001 is an antisense oligonucleotide therapeutic product that targets CPS1 enzyme, responsible for ammonia to urea conversion. It enhances the CPS1 gene expression, increasing CPS1 protein levels
Ref: CAMP4 Therapeutics | Image: CAMP4 Therapeutics
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
