Think of CRISPR-Cas as a pair of tiny, molecular scissors. It is a tool scientists use to cut and edit DNA, the building blocks of life. CRISPR-Cas can help modify genomes to prevent and cure diseases. Scientists are using it to study and treat diseases like cancer and genetic disorders. As researchers and industries explore its vast potential, the patent landscape surrounding the technology has become increasingly complex. This article discusses the fundamentals of CRISPR-Cas, its wide-ranging applications in medicine, and the innovation that can help shape the future of gene editing.

Introduction

CRISPR-Cas technology has opened remarkable possibilities in gene editing and its potential impact in the field of medicine and healthcare. CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, functions as a genetic scissor that can enable precise modifications to DNA.

This technology’s potential is evident in the accolades it has received, including the 2020 Nobel Prize in Chemistry awarded to researchers Emmanuelle Charpentier and Jennifer Doudna for discovering CRISPR-Cas9. Estimates suggest that investments worth US $3.78 billion to date have been made in CRISPR startups.

As CRISPR-Cas continues to revolutionize the life sciences, it also presents a complex and ever-expanding IP landscape. With over 11,000 families of CRISPR-related patents already filed, the question of who holds the rights to this groundbreaking technology is both crucial and contentious.

This article explores the fundamentals of CRISPR-Cas, its wide-ranging applications, the ethical challenges it presents, and the intricate legal disputes that define its patent landscape.

The Building Blocks

CRISPR-Cas technology is derived from an ancient defense mechanism found in prokaryotes, which are single-celled organisms without a nucleus. Bacteria is a common type of prokaryote.  

The CRISPR technology functions as an immune response, where bacteria deploy CRISPR-associated (“Cas”) proteins to target and cleave the DNA of invading viruses. By harnessing this mechanism, scientists have been able to engineer the CRISPR-Cas system to perform precise, targeted cuts in the DNA of more complex organisms, including plants and animals.

Among the various Cas proteins identified, CRISPR-Cas9 is most widely used and recognized. Its ability to edit the genome with high precision has made it an invaluable tool for a range of applications, from basic research to therapeutic development.

For instance, in 2020, a patient suffering from Leber congenital amaurosis, which is a genetic disorder that leads to blindness, became the first to receive an in-vivo CRISPR-Cas9 therapy. This breakthrough marked a significant milestone in the application of CRISPR-Cas technology, signaling its potential to treat a wide array of genetic conditions.

Beyond CRISPR-Cas9

While CRISPR-Cas9 remains the most prominent tool in gene editing, the field is rapidly advancing with the discovery and development of new CRISPR-associated enzymes. These include Cas12, Cas13, CasX, and CasY, with each offering unique capabilities. For example, Cas12 and Cas13 are known for their ability to target single-stranded RNA viruses like coronaviruses, opening up possibilities for combating RNA viruses like SARS-CoV-2, the virus responsible for COVID-19.

The development of these new tools expands the potential applications of CRISPR in the field of medicine. Research indicates that one of its most exciting applications is in the field of gene therapy where it could be used to correct genetic mutations responsible for hereditary diseases.

Researchers are also currently exploring the use of CRISPR to treat conditions such as cystic fibrosis, Duchenne muscular dystrophy, hemophilia, and dermatology related issues. By directly editing the faulty genes that cause these diseases, CRISPR may offer the possibility of permanent cure rather than symptomatic treatments.

Cancer research is another area where CRISPR is making significant strides. Scientists are using CRISPR to engineer T-cells, a type of immune cell, to better recognize and attack cancer cells. This approach, known as CAR-T cell therapy, has already shown promise in treating certain types of blood cancers and is now being adapted for solid tumors. This therapy is an immunotherapy for cancer. It uses the body’s own immune defenses as an ally in the fight against cancer. 

Moreover, CRISPR is being explored as a tool for combating infectious diseases. By targeting and disabling viral DNA within infected cells, CRISPR could potentially respond to chronic viral infections such as HIV and hepatitis B.

Intellectual property and CRISPR technology

The rapid development of CRISPR-Cas technology has led to a complex and competitive patent landscape. A high-profile litigation between two major groups is central to the contour of the patent landscape: the University of California, Berkeley, the University of Vienna, and Dr. Emmanuelle Charpentier (collectively known as CVC) on one side, and the Broad Institute, affiliated with Harvard and MIT, on the other. Both groups claim ownership of the fundamental inventions related to CRISPR-Cas9, particularly its use in eukaryotic cells, which includes plants and animals.

The dispute traces back to 2012 when Dr. Doudna and Dr. Charpentier first published their findings on the CRISPR-Cas9 system in prokaryotes and subsequently filed for a patent. Several months later, Dr. Feng Zhang of the Broad Institute published his research demonstrating the use of CRISPR-Cas9 in eukaryotic cells and filed for a patent as well.

The Broad Institute’s patent was granted in April 2014, leading the CVC group to challenge the decision. The United States Patent and Trademark Office (USPTO) ruled in favor of the Broad Institute, a decision later upheld by the Federal Circuit. A second patent interference dispute followed in 2019. In 2022, the USPTO reaffirmed the Broad Institute’s priority over the use of CRISPR-Cas9 in eukaryotic cells. The CVC group has since appealed this decision, and the matter is currently pending with the court. Moreover, the dispute has extended beyond the United States, with multiple proceedings underway in Europe.

The determination of ownership plays a crucial role in driving innovation. Without clear ownership, it becomes difficult to identify who holds the rights to license a technology, creating uncertainty that can hinder further development and investment. In this sense, litigations that clarify or resolve disputes over ownership are essential. They ensure that innovators can confidently access and build upon existing technologies, fostering a more dynamic and productive field of innovation.

In addition to litigation, alternative dispute resolution (ADR) procedures, such as mediation and arbitration, may offer a flexible and efficient alternative for resolving complex patent disputes. The WIPO Arbitration and Mediation Center provides tailored ADR services, including access to expert arbitrators and mediators with technical and legal expertise in the life sciences sector. As the CRISPR patent landscape evolves, these specialized ADR options may allow parties to resolve disputes in a confidential and less adversarial setting, helping to preserve relationships and promote ongoing collaboration in dynamic industries like biotechnology.

What’s next

For companies and researchers looking to innovate in the CRISPR space, navigating this dense and ever-evolving IP terrain can be a challenge. Ensuring freedom to operate (FTO) requires careful consideration of existing patents and potential licensing requirements, which may vary depending on the intended application of the technology. This complexity is compounded by the fact that different entities hold patents for different aspects of CRISPR-Cas technology, often requiring multiple licenses to operate within a single field.

That being said, CRISPR technology holds great potential to revolutionize healthcare by providing precise tools for gene editing. It offers the ability to correct genetic mutations that cause a wide range of diseases, including inherited disorders like cystic fibrosis and sickle cell anemia. Beyond treating genetic diseases, CRISPR could be used to engineer immune cells to better fight cancer, develop new antibiotics to combat drug-resistant infections, and even create more efficient gene therapies. Its versatility and precision make it one of the most promising advancements in modern medicine, with the potential to reshape how we prevent, treat, and cure diseases.