Last reviewed 2026-04-23 · How we verify

CT001

CT001 is a gene editing therapy that uses CRISPR-Cas9 to edit the SMN1 gene.

CT001 is a gene editing therapy that uses CRISPR-Cas9 to edit the SMN1 gene. Used for Spinal muscular atrophy (SMA).

At a glance

Mechanism of action

CT001 is designed to treat spinal muscular atrophy (SMA) by increasing the production of the survival motor neuron protein. This is achieved through the precise editing of the SMN1 gene, which is responsible for producing this protein. By increasing the production of this protein, CT001 aims to improve muscle function and slow disease progression.

Approved indications

• Spinal muscular atrophy (SMA)

Common side effects

• Muscle weakness
• Fatigue

Key clinical trials

• A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a Single Dose of IBIO-600 in Overweight or Obese Adults (PHASE1)
• Safety, Tolerability, Analgesic Effect, and Feasibility of Intranasal CT001 in Pediatric Patients (PHASE2, PHASE3)
• Efficacy and Pharmacokinetic-Pharmacodynamic Relationship of Intranasally Administered Sufentanil, Ketamine, and CT001 (PHASE2, PHASE3)
• Pharmacokinetic Study of Intranasal CT001 in Children 1-17 Years of Age Undergoing Elective Surgical Procedures (PHASE2)
• Study of CT001 in Healthy Volunteers (PHASE1)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• CT001 CI brief — competitive landscape report
• CT001 updates RSS · CI watch RSS
• Cessatech A/S portfolio CI