In 2015, CRISPR Therapeutics had a strategic research partnership with Vertex. The CTX001 ex vivo CRISPR gene-edited therapy lead program for sickle cell disease and β-thalassemia is the product of that partnership.

On April 20, CRISPR Therapeutics amended its collaboration with Vertex Pharmaceuticals to manufacture and commercial CTX-001. Under the new arrangement, instead of receiving a small percentage of revenue from sales, CRISPR will receive $900 million in upfront cash as capital. Once regulators approve CTX-001, Vertex will grant CRISPR $275 million in additional cash to the original collaboration agreement.

A Leader in Investigational Medicine

According to CRISPR, the CLIMB THAL-111 study, which targeted transfusion-dependent beta-thalassemia patients, yielded positive findings in December. CRISPR is a leader in gene therapy with proprietary licenses for CRISPR Technology in terms of intellectual property. It's a pioneer in Cas9 gene editing, along with a few other biotech firms. Preclinical trials indicate that CRISPR technology is better and more efficient than the now-suspended Zyneteglo's Lentiviral Vector gene therapies and Adeno-Associated Vector therapies used by Luxturna.

The Risk of the Unknown

CRISPR Therapeutics is not without some risks. While the benefits of increased accuracy and precision are there, concerns about excessive evolution to disease-causing bacteria that could become immune to CRISPR drugs are possible.

In addition, Biotech stocks are frequently subject to unpredictable binary events, including clinical trial outcomes and U.S. FDA drug approval decisions. As such, Biotech companies could see a significant change in their stock price if they experience adverse side effects or less-than-expected efficacy from drug trials. There are some concerns with the CRISPR technology since there have not been any long-term studies to show how safe or effective this therapy will be.

The Intersection of Opportunity

CRISPR Therapeutics' market success is coming in part from anticipating the future of healthcare. Gene editing and gene therapy are rapidly advancing, and CRISPR Therapeutics has leaped ahead to lead this emerging field.

Will their unique advantage around CRISPR technology yield the results of providing new treatments for genetic disorders such as cystic fibrosis and Duchenne muscular dystrophy? It's too early to tell, but CRISPR Therapeutics has the opportunity to make a significant impact on people's lives.