Top 11 CRISPR Stocks to Invest In

2. Intellia Therapeutics, Inc. (NASDAQ:NTLA) 

Number of Hedge Fund Holders: 30

Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a biotech company at the forefront of CRISPR-Cas9 gene-editing technology, working on innovative therapies for both in vivo and ex vivo applications. Its leading program, NTLA-2001, is aimed at treating transthyretin amyloidosis, a currently incurable condition. Another promising treatment in its pipeline, NTLA-2002, targets hereditary angioedema (HAE) and has shown promising results in early clinical trials.

BMO Capital remains optimistic about Intellia’s prospects, maintaining an Outperform rating and a $70 price target in October. This confidence is bolstered by the Phase II trial results for NTLA-2002, which demonstrated an 81% reduction in attack rates compared to placebo over 5 to 16 weeks, with 73% of patients remaining attack-free. However, some concerns were noted: NTLA-2002 didn’t meet expectations of having over 80% of patients attack-free, and the attack-free rate appeared to decline as the trial progressed, a trend observed in other HAE trials as well.

In Q3, Intellia Therapeutics, Inc. (NASDAQ:NTLA) reported several milestones: FDA clearance for the Phase III MAGNITUDE-2 trial of nexiguran ziclumeran for ATTR amyloidosis, strong enrollment in the MAGNITUDE study, and active screening for the HAELO Phase III trial for HAE. The company ended the quarter with $944.7 million in cash, a solid runway extending into late 2026. Financially, it recorded $9.1 million in collaboration revenue, $123.4 million in R&D expenses, and a net loss of $135.7 million.