NB-101
Alpha-1 antitrypsin deficiency (AATD)
Pre-clinicalActive
Key Facts
Indication
Alpha-1 antitrypsin deficiency (AATD)
Phase
Pre-clinical
Status
Active
Company
About Nerai Biosciences
Nerai Biosciences is a private, pre-clinical stage biotech founded in 2021 in Zurich, Switzerland. The company's core innovation is the MORPHEME platform, which integrates high-throughput directed evolution, functional screening, and machine learning to engineer novel CRISPR enzymes with superior specificity and activity. Nerai is advancing a proprietary pipeline of genome editing therapies for rare genetic diseases, with its lead program for Citrullinemia type 1 in in vivo lead optimization. The company is backed by non-dilutive grants and awards, positioning it to address previously inaccessible genetic mutations.
View full company profileTherapeutic Areas
Other Alpha-1 antitrypsin deficiency (AATD) Drugs
| Drug | Company | Phase |
|---|---|---|
| Unnamed AATD Program | AlveoGene | Pre-clinical |
| Undisclosed | GondolaBio | IND Enabling |
| TSRA-196 | Tessera Therapeutics | Phase 1 |
| BEAM-302 (for Beam Therapeutics) | Richmond Pharmacology | Phase 1/2 |
| KB408 | Krystal Biotech | Preclinical |
| Alpha-1 Antitrypsin (AAT) | Grifols | Marketed |
| BEAM-302 | Beam Therapeutics | Phase 1/2 |
| GalNAc Program | Korro Bio | Preclinical |