Intellia has submitted its first Clinical Trial Application (CTA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1 study of NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR). Intellia is submitting additional regulatory applications to enable enrollment in other countries as part of its global clinical development plans.
NTLA-2001 is a potential single-course therapy for patients living with ATTR. Pending CTA authorization and subject to the impact of COVID-19, Intellia is planning to start its first clinical trial in ATTR patients by the end of 2020. This is the first time a CRISPR/Cas9 genome editing treatment will be delivered through a vein of the human body to edit genes.
This Phase 1 trial will be a single-ascending dose study for ATTR patients, meaning that prior to going to the next dose level, available data from all subjects that have been dosed with NTLA-2001 will be reviewed to determine if a higher dose should be given to the next group of participants.
This trial is intended to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001. In other words, the purpose of this study is to test the safety and how well patients tolerate NTLA-2001.
More information about Intellia’s Phase 1 study of NTLA-2001 coming soon.