crispr_img_1A contentious patent battle has continued to rage between the Broad Institute at Harvard/MIT and the University of California (UC). UC is challenging 13 patents related to CRISPR gene editing technology that are currently held by the Broad Institute. The basis of the challenge lies in explaining the potential influence that the work of Jennifer Doudna (UC Berkeley) and Emanuelle Charpentier (then at Umeå University) had on Feng Zhang’s (Broad Institute) later work, and whether his work was an obvious development beyond that of Doudna/ Charpentier, or whether it has the earliest priority claim. Although the patent battle remains active with an uncertain outcome, Doudna, Charpentier, and Zhang remain key players with regards to both the patents and research advances/ startup companies related to the technology.

As the patent battle for CRISPR gene editing technology wages on, it has been easy to overlook other clinical and therapeutic advances that these companies are researching and pioneering at a rapid pace. Although early investigations have focused primarily on oncology/ cancer treatments, many expect the field of CRISPR-based therapeutics to progress into treating rare and orphan diseases, many of which have well-understood pathologies and disease mechanisms, but that currently lack effective treatments. With regards to these diseases, there is huge promise for a lifelong cure after just a single treatment. Understandably, this is extremely attractive to patients. The development of novel treatments and therapeutic approaches promises to reveal clear opportunities for patent-eligible therapeutics and other related findings, even in light of the current CRISPR patent landscape.

CRISPR (clustered regularly interspaced short palindromic repeats) was originally discovered and described as a bacterial immune system, serving to fend off viral infections by bacteriophages. The real value of the technology, however, was soon recognized as a gene editing technology with clear promise not only for basic science applications, but also for its therapeutic potential. CRISPR-related research and applications efforts have exploded in recent years, resulting in several companies seeking to develop technologies that focus on CRISPR-based therapeutics. Included are Editas, CRISPR Therapeutics, Caribou Biosciences, and Intellia Therapeutics.

Editas, a publically traded company as of early 2016, is not only focusing on engineering various aspects of the CRISPR system to achieve more precise directed genome targeting, but also modalities and methods of delivery including viral vectors, nanoparticles, and engineered cells. Intellia Therapeutics, another company that went public in 2016, is also focused on better engineering CRISPR technology for more effective and targeted disease treatment. Intellia is targeting both in vivo and ex vivo therapies. Although Editas and Intellia have experienced dips in their stock prices since their IPOs, it is possible that these price drops are indicative of the movement of the larger market, and in particular, biotechnology trading patterns this year. It will be interesting to track the stock performance of CRISPR Therapeutics, which has just recently gone public in late October, as they continue to develop novel therapies for beta-thalassemia, sickle cell disease, severe combined immunodeficiency, hemophilia, and cystic fibrosis, among others.

Although promising, many questions do remain with regard to CRISPR-based therapies. For example, it is as of yet unclear whether patients receiving a course of therapy could develop an immune response against the introduced CRISPR therapeutic, rendering it ineffective and causing potential harm to the patient. Off target editing effects are also a concern with CRISPR-based therapeutics. It will be essential for those developing technologies and therapies to show that off target editing effects are virtually absent, and that there are no detectable modifications that could result in heritable changes from generation to generation. Although CRISPR-based technologies have shown a decrease in off target effects when compared to earlier gene editing technologies, many still report the present of off target effects, which likely will be unacceptable in treatments. There are also many regulatory and safety concerns that must be considered as these technologies and therapies are being developed, including the potential for non-therapeutic use, which could theoretically result in parents selecting both physical and non-physical traits for their unborn children.

In assessing the current landscape of patents in this area, it is readily apparent that the space is largely dominated by the initial patents that were filed for CRISPR technologies by Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang, and that research being done by the previously mentioned companies and others has been governed by the granting of both exclusive and non-exclusive licenses. As the patent battle for CRISPR technologies continues, we eagerly anticipate inventors being required to approach the subject of patent-eligibility with regards to CRISPR in a new light- one that not only allows for the patenting of novel therapeutics, methodologies, and advances, but also remains flexible in light of the pending ruling on the current Broad Institute/ UC Berkeley case.

– Nicholas Vincent and Anthony D. Sabatelli, PhD, JD

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