10 Stocks That Will Change the World

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

Number of Hedge Fund Holders In Q1 2024: 29

Intellia Therapeutics, Inc. (NASDAQ:NTLA) is another gene editing company. The firm develops treatments for hemophilia, lung disease, angioedema, and other ailments. Intellia Therapeutics, Inc. (NASDAQ:NTLA) is currently making rapid progress in its angioedema treatment, and the firm is slated to start an all important phase three clinical trial in the current half of this year. It is also testing its cardiomyopathy treatment, which creates additional potential tailwinds for the stock. Intellia Therapeutics, Inc. (NASDAQ:NTLA)’s angioedema treatment trial also expands the target base to women of childbearing age, which means that the market for the drug will be larger than other products from rivals. Gene editing is a technology that can change the world, and Intellia Therapeutics, Inc. (NASDAQ:NTLA) might benefit from it if it manages to successfully execute its product pipeline and keep other troubles at bay.

Intellia Therapeutics, Inc. (NASDAQ:NTLA)’s management shared details for its treatments during the Q2 2024 earnings call when it shared:

“A single 25- or 50-milligram dose of NTLA-2002 led to deep reductions in attacks for patients with HAE. No new safety findings were observed. We have now selected the 50-milligram dose to advance into the global Phase III trial because we saw greater calotrene reduction and, importantly, a higher number of patients who achieved complete elimination of attacks compared to the 25-milligram dose cohort, which is consistent with the prior Phase I results. In addition to the positive results, we recently completed a successful end of Phase II meeting with the FDA. We believe we have completed addressing their questions and have a clear understanding of the path forward.

We are on track to begin the Phase III trial in the second half of this year and, if positive, plan to submit the BLA for NTLA-2002 in 2026. We anticipate that NTLA-2002 will be the first in vivo gene-editing therapy to come to market and, most importantly, we strongly believe it will reset the standard of care for HAE. Switching now to NTLA-2001 for the treatment of ATTR amyloidosis, the drug now has a new name, Nexiguran Ziclumeran, or NEX-Z for short. Recent competitor data confirmed our longstanding hypothesis that TTR reduction leads to a clinically meaningful improvement in patients with cardiomyopathy. This is an important advancement in the field’s understanding of the disease, and this result further increases our confidence that NEX-Z could lead to even better clinical outcomes for patients.”