In the past few decades, medical attention began to shift from developing pharmaceuticals that target proteins to developing techniques to directly editing the genes that code for proteins.
One particular gene-editing technique holds great potential for successfully manipulating genes, but it is currently the subject of a patent court aimed at identifying its rightful owner.
This system is called CRISPR, which stands for clustered regularly interspaced short palindromic repeats. Dr. Jennifer Doudna is a researcher at University of California, Berkley who was the first to publish a paper describing the mechanism of the CRISPR system in 2013, and first to file a patent application for her finding.
However, Massachusetts Institute of Technology researcher Feng Zhang also focused on CRISPR in his own research and filed an expedited patent request later that same year.
Zhang was awarded a patent in 2014 and is legally considered the intellectual property owner of CRISPR, but the case is undergoing further review.
CRISPR works with a family of proteins named Cas, which stands for “CRISPR associated proteins,” to identify and cut DNA at specific sequences, and then insert other sequences at the same location. This complex system naturally occurs in prokaryotes as an immune system defense mechanism against invading DNA such as that from a bacteriophage, a virus that infects bacteria.
While studying bacterial immunity, Doudna and her team thought that the Cas9 protein might have the potential to edit genomes of other organisms, so they developed a system to do so. Zhang and his group were first to generate successful results in by using Doudna’s CRISPR system to edit genomes of human cell cultures.
This new CRISPR system uses two main components: a protein, named Cas9, and an RNA strand, called single-guide RNA (sgRNA). The sgRNA contains the target sequence to be edited, and directs Cas9 to the desired site to where it can act as an endonuclease and physically cut DNA’s double helix. Cas9 can then insert a different DNA sequence, thereby rewriting a harmful genetic mutation or excising a malignant defect.
Two patent law prerequisites play a significant role in this patent court: the “written description requirement” and the “enablement requirement.”
The written description requirement states that patent applicants must provide a detailed explanation of the invention to prove that they currently possess that invention; the enablement requirement states that the applicant must provide written instructions that could enable a person skillful in the particular field to use or carry out his or her invention.
While both Doudna and Zhang have since published many papers that discuss CRISPR in detail, Zhang seems to have an advantage because he was first to publish a paper that described how to use CRISPR in different organisms.
CRISPR’s patent court illustrates the fierce competition inherent in innovative fields, but regardless of CRISPR’s patent court outcome, this technology will have an immense impact in numerous areas, from agriculture to medicine to finance.
CRISPR has already been shown to be effective in instilling fungal resistance in species of wheat, developing abnormally muscular livestock and manipulating human embryo genomes, showing the potential to eradicate harmful disease.
However, this technology’s full potential has yet to be realized; while millions of dollars of funding have been distributed to institutions for CRISPR research, analysts estimate the technology to generate a market of over $5 billion by 2020.