What happened

Shares of Crispr Therapeutics (NASDAQ:CRSP) rose 17% last month, according to data provided by S&P Global Market Intelligence. The pioneer in gene-based therapy announced third-quarter 2018 operating results. As a development-stage company, the only financial metric that really matters to investors is the cash balance, which hit a record $487 million at the end of September following a massive share offering. 

Every penny will be needed to hit the ground running in developing the company's clinical pipeline. Crispr Therapeutics and partner Vertex Pharmaceuticals announced in October that the U.S. Food and Drug Administration had lifted the clinical hold on CTX001 for sickle cell disease. That will allow the drug candidate to begin enrollment in a phase 1/2 trial before the end of 2018. That's not the only thing investors are excited about.

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So what

Crispr Therapeutics has done well maintaining its lead over peers Editas Medicine and Intellia Therapeutics. While there isn't direct competition among the trio's lead drug candidates, Crispr Therapeutics was the first to initiate a clinical trial for a CRISPR-based therapy.

In fact, CTX001 is now being investigated in phase 1/2 trials for beta-thalassemia and sickle cell disease. Each trial will dose two patients and, pending the data collected from those efforts, will expand to 45 patients. That's when things could really get interesting for investors hoping to cash in on the promise of gene editing.

The company is also toiling away at pre-clinical efforts for its wholly owned next-generation CAR-T therapeutic candidates, which could offer improved safety and efficacy over current immunotherapies aimed at solid-tumor cancers. Additionally, Crispr Therapeutics is working with ViaCyte to develop engineered stem cells to treat -- and perhaps cure -- diabetes.

Now what

The leading gene-editing stocks have jumped all over the place in the last year. Despite the volatility, shares of Crispr Therapeutics have delivered an 85% gain in that span and are now up 49% since the beginning of 2018. Investors who believe first-generation gene-editing products will deliver on their promise certainly cannot complain, but there will be more patience required in the months and years ahead.

This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.