Based on the Nobel-prize winning genome editing tool CRISPR-Cas9, NTLA-2001 is an investigational treatment for patients with ATTR amyloidosis, a rare and fatal disease that leads to neuropathy and heart failure. NTLA-2001 aims to permanently inactivate the target gene and prevents the production of offending proteins. NTLA-2001 holds the potential to halt and reverse the disease following a single dose.
Richmond Pharmacology was the first CRO globally to dose a patient in a landmark CRISPR-Cas9 clinical trial of Intellia Therapeutics’ asset NTLA-2001. The primary objectives of this on-going trial were to assess safety and pharmacodynamics and determine the correct dose to block the production of faulty ATTR.
Initial results are positive and indicate a reduction in the faulty TTR protein after a single infusion, with mild adverse events. The patients participating in this study will be observed for three years by Professor Julian Gillmore of the National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital.
Read the supporting publication in The New England Journal of Medicine: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
ExoIL-12 has been developed to treat solid tumours that contain T cells and NK cells. ExoIL-12 controls the dose of IL-12 and targets the tumour micro-environment without causing systemic drug exposure and preventing serious adverse events. This enables safe application in patients and highly accurate targeting to the affected area.
This is an important breakthrough as previous trials by other drug development companies showed detectable systemic exposure using IL-12. This trial was the first attempt using the Exo therapy in a Phase I trial.
The phase I trial was conducted in a period of five months, the first subject was dosed in September 2020 and the last subject visited in January 2021. We maintained a 100% retention rate throughout the pandemic and all the sponsor’s timelines were met.
Richmond Pharmacology has a team of experts with extensive knowledge of clinical research trials.
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