On June 26, Intellia Therapeutics and Regeneron issued a press release detailing efficacy and safety data for the momentous first use of in vivo clustered regularly interspaced short palindromic repeats (CRISPR) genome editing in humans in Phase I clinical trials. The investigational CRISPR therapy, NTLA-2001, caused deep reductions in a disease-causing protein, misfolded transthyretin (TTR), after a single infusion. The novel therapeutic approach is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. Currently, the only available treatment options for ATTR are chronic treatments. This landmark clinical data is exciting for patients with ATTR and for the field of genomics as a whole. Shares of the three leading companies in the CRISPR-based therapeutics space (Intellia Therapeutics Inc, NTLA: 50+%, CRISPR Therapeutics AG, CRSP: 15+% and Editas Medicine Inc, EDIT: 20+%) soared after the data were released, reflecting the market’s perception that the recent data have validated the therapeutic potential of the technology. In a March 12 press release, GlobalData profiled the pipeline of these three leading companies and highlighted the disruptive nature of the CRISPR technology.

An interim readout of an ongoing Phase I trial found that a single 0.3mg/kg dose of NTLA-2001 led to an 87% mean reduction in serum TTR, with a maximum 96% serum TTR reduction by day 28, and showed a dose-dependent response. No serious adverse events were recorded in the first six patients by day 28, with patients only experiencing infusion-related reactions to the intervention. ATTR causes an accumulation of misfolded proteins, which form deposits in the nerves, heart, kidney, and eyes. The typical life expectancy of patients with ATTR is 2–15 years from the onset of symptoms and there are approximately 50,000 hereditary ATTR cases globally, highlighting a large addressable patient population. Marketed treatment options do exist in this space and can lead to a mean reduction of serum TTR of 80%, but typically involve an infusion every three weeks. NTLA-2001 has the opportunity to disrupt the market by introducing a more efficacious, convenient treatment that tackles the condition at the DNA level, thus addressing the origin of the hereditary disorder. Intellia is currently in the dose-optimising period of NTLA-2001’s clinical development, suggesting that further efficacy and even a cure could be achieved in the future.

Notably, this study had a small sample size of six patients and further validation is required. Specifically, determining whether CRISPR genome editing can incur abnormal genetic insertions and deletions in vitro. These are well known to be the basis of cancerous growth, emphasising the need to monitor patients over the long term. Furthermore, the genetic deletion of TTR may cause defects in vitamin transfer, requiring patients to take vitamin supplements indefinitely.

However, this efficacy and safety data has shown a clear indication of the power of CRISPR-based therapeutics and their role in the future of healthcare. Further catalysts highlighted in GlobalData’s Catalyst Calendar highlight key events that will further reveal the prospects for the leading CRISPR companies, including expected read-outs for CRISPR Therapeutics’ allogenic chimeric antigen receptor T (CAR-T) cell programme later this year.

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