Approval Probability
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CTX460 · 1 trial · 1 indication
Incidence of DLTs and frequency of AEs
Absolute blood levels of total AAT at Month 6
| Arm | Type | Description |
|---|---|---|
| Phase 1 | EXPERIMENTAL | Phase 1 begins with dose escalation based on a single infusion of CTX460. |
| Phase 2 | EXPERIMENTAL | Recommended Phase 2 dose of CTX460. |
| Name | Type | Description |
|---|---|---|
| CTX460 | DRUG | CTX460 is a lipid nanoparticle (LNP) formulation of synthetic nucleotide template polymerase (SyNTase) editor for in vivo gene editing of the serine protease inhibitor A1 (SERPINA1) gene in adults with emphysema associated with severe alpha-1 antitrypsin deficiency (AATD). |
Inclusion Criteria: 1. Age: ≥18 and ≤75 years. 2. Able to provide written informed consent. 3. Diagnosis of AATD with PiZZ homozygous genotype. 4. Serum total AAT level \<11µM in the absence of augmentation. 5. Emphysema on a CT scan at screening. 6. A postbronchodilator forced expiratory volume in...
CTX460 is an investigational small molecule developed by CRISPR Therapeutics AG for alpha-1 antitrypsin deficiency (AATD), a genetic condition that can lead to emphysema. It is designed to target SERPINA1, the gene that encodes the alpha-1 antitrypsin protein. CTX460 is currently in Phase 1 clinical development and has not been approved by the FDA.
CTX460 is being developed for adults with emphysema associated with severe alpha-1 antitrypsin deficiency (AATD). AATD is a genetic disorder in which the liver produces insufficient or dysfunctional alpha-1 antitrypsin protein, leaving the lungs vulnerable to damage. CTX460 is investigational and its safety and efficacy in this indication have not been established.
CTX460 targets SERPINA1, the gene that encodes the alpha-1 antitrypsin protein. In alpha-1 antitrypsin deficiency, mutations in SERPINA1 reduce or impair production of functional alpha-1 antitrypsin, which normally protects lung tissue. By acting on SERPINA1, CTX460 is intended to address the underlying genetic cause of the disease.
CTX460 is being developed by CRISPR Therapeutics AG, a biotechnology company whose common stock trades on the Nasdaq under the ticker symbol CRSP. CRISPR Therapeutics is the sponsor of the clinical program evaluating CTX460 in adults with emphysema associated with severe alpha-1 antitrypsin deficiency.
CTX460 is in Phase 1 clinical development. It is an investigational small molecule and has not been approved by the FDA for any indication. The Phase 1 program is evaluating the drug in adults with emphysema associated with severe alpha-1 antitrypsin deficiency, and no trials of CTX460 have been completed to date.
CTX460 is being evaluated in one clinical trial, NCT07850076, titled a trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of CTX460 in adults with emphysema associated with severe alpha-1 antitrypsin deficiency (AATD). The Phase 1 study is controlled, is not yet recruiting, and is planned to enroll 68 participants aged 18 years and older.