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 and 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 with speed and efficiency.

Our 10-year collaboration with the National Amyloidosis Centre has positioned us as the largest recruiter for gene editing trials like Intellia's and we have created a new, more efficient, model for identifying potential patients.

This, combined with the fact that we have a large team of highly trained experts able to treat a high volume of patients, makes us a global leader.