Vertex Pharmaceuticals Inc and CRISPR Therapeutics AG have gained a world-first regulatory approval for the use of a CRISPR-based gene editing therapy in patients, as the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorisation for CASGEVY.[1],[2]
CASGEVY is a therapy designed to treat sickle-cell disease and inherited blood disorders, and works by isolating and editing a patient’s bone marrow-derived stem cells before infusing the genetically altered cells back into the patient. The treatment has been found to restore haemoglobin production in most cases, with no significant safety concerns.
The therapy involves using CRISPR/Cas9 technology to edit genes that control haemoglobin levels. CRISPR, which stands for “Clustered Regularly Interspaced Short Palindromic Repeats”, and Cas9, which is an endonuclease protein that cuts DNA, are both components of certain bacterial adaptive immune systems that can be modified and targeted to certain cells and DNA sequences (including in human cells) to perform a variety of gene editing functions. The technology was so groundbreaking, its discoverers were awarded the 2020 Nobel Prize in Chemistry.
CRISPR Therapeutics was co-founded by Emmanuelle Charpentier in 2013 and entered into collaborations with Vertex in 2015 to explore the development of CRISPR/Cas9 technology for novel therapeutics. Charpentier, in conjunction with the University of California/Berkeley and the University of Vienna (collectively, “CVC”) was one of the first groups to seek patent protection for CRISPR/Cas9 systems after their discovery in 2012. Within months, patent applications were also filed covering various aspects of CRISPR/Cas9 technology by the Broad Institute, Harvard University, and MIT (collectively, “Broad”), ToolGen Inc, and Sigma-Aldrich.
The majority of granted patents for CRISPR/Cas9 are currently held by CVC and Broad, but given the huge therapeutic potential and therefore commercial interest in CRISPR/Cas9, there have been a number of high profile disputes between these and other patent owners or would-be rights holders. For example, the most recent US Patent Trial and Appeal Board decision that Broad retain its IP over the use of CRISPR/Cas9 in eukaryotes[3] is currently under appeal by CVC to the US Federal Circuit Court, and US interference proceedings brought by ToolGen and Sigma-Aldrich are also pending. In Europe, an invalid priority claim resulted in revocation one of Broad’s key patents.[4] Even in Australia, we have seen several oppositions and most recently a Federal Court action in the CRISPR/Cas9 field.[5]
CASGEVY is no doubt the first of many CRISPR therapeutics to join the global market. As they do, each party will have more to gain from retaining as much ownership of the foundational IP as possible, and any wounds inflicted by losing ground will sting a little bit more.
[1] Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia | Vertex Pharmaceuticals (vrtx.com)
[2] Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia | CRISPR Therapeutics (crisprtx.com)
[3] USPTO Interference No. 106,116
[5] ToolGen Incorporated v Fisher (No 2) [2023] FCA 794