Gene Editing & Gene Silencing

Spearheading First in human gene therapy research

In 2020, we made history as the first to dose a patient using an in-vivo CRISPR-Cas9 therapy, just a year before it was awarded the Nobel Prize.

Since then, we've continued to pioneer first in human gene therapy research, dosing over 100 patients across multiple gene therapy trials, setting a global benchmark for clinical excellence.

First dosing with an in-vivo CRISPR-Cas9 therapy

In 2020, Richmond Pharmacology conducted the world's first trial involving a CRISPR-Cas9 therapy to treat ATTR amyloidosis, a rare and life-threatening disease that causes heart failure and neuropathy. This breakthrough therapy, developed by Intellia Therapeutics, inactivates the gene responsible for harmful amyloid protein production. Just a single dose holds the potential to halt and even reverse the disease’s progression.

Our strong relationships with NHS clinicians and leading specialist centres allow us to recruit and enrol patients expediently/efficiently/seamlessly, ensuring the successful recruitment of trials. Our collaboration with the National Amyloidosis Centre has positioned us globally? As the largest recruiter for trials like Intellia's harnessing effective identification of potential patients and the team and facility to recruit and treat a high volume of patients.

Pioneering the Largest CRISPR Trials

In March 2024, Richmond Pharmacology was the first to dose a patient in the world’s largest Phase 3 trial of a CRISPR-Cas9 therapy to date. As we continue to enrol patients in this landmark trial, we are setting the benchmark for integrate identification, recruitment and enrolment of clinical trials worldwide.

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