Richmond CEO Presents Landmark Data on Repeat-Dose Gene Editing

A presentation by Richmond CEO Jorg Taubel on repeat dose gene editing has received widespread media coverage. 

At the 2024 Peripheral Nerve Society (PNS) Annual Meeting, held in Montreal, Canada, Richmond Pharmacology CEO Dr Jorg Taubel unveiled groundbreaking findings that mark a milestone in gene-editing therapies. The presentation focused on the first-ever study investigating the safety and efficacy of a repeat dose of Intellia Therapeutics' CRISPR-based gene-editing therapy, NTLA-2001.

This pioneering research, conducted with Intellia Therapeutics, represents a major advancement in the field of genetic medicine. The therapy targets hereditary ATTR amyloidosis (hATTR), a progressive and often life-threatening disease caused by misfolded proteins that accumulate in tissues such as the nerves and heart. NTLA-2001 is designed to correct the underlying genetic mutation through in vivo CRISPR/Cas9-mediated editing, potentially offering long-term therapeutic effects.

A New Era for Gene Editing

In gene therapy, single doses are often the standard approach. However, Dr Taubel’s presentation detailed a crucial evolution: patients who had initially received a low dose were later re-administered a higher therapeutic dose once safety and efficacy had been established. This repeat-dosing concept is a novel strategy in the gene-editing space and highlights the potential for increased therapeutic benefits through dose optimization.

The repeat-dose data revealed encouraging safety and efficacy profiles, opening new doors for future gene-editing therapies. "This study is the first of its kind and could redefine how we approach gene editing in complex genetic disorders like hATTR," Dr Taubel explained.

Global Media Coverage

The importance of this proof-of-concept study attracted significant media attention, with coverage by notable publications such as by Fierce Biotech, Yahoo Finance, Rare Disease Advisor, FAP News Today and CGT Live. These outlets highlighted the transformative potential of repeat-dose gene editing and its implications for future therapeutic applications. The news of this first ever study into repeat gene editing was covered 

Implications for Future Therapies

The NTLA-2001 study marks an important step toward making gene editing a feasible, long-term treatment for patients with complex genetic diseases. By demonstrating the safety of repeat dosing, this research could encourage further exploration of multiple dosing regimens for other genetic therapies.

Dr Taubel’s insights into repeat-dose gene editing could pave the way for expanded indications, making treatments more effective for a wider range of patients. The ability to revisit and adjust dosing may allow more precise control of gene-editing outcomes, especially in progressive conditions that may require ongoing intervention.

Read the Intellia Therapeutics press release here.