One Year On: The Future of CRISPR is Now
As I prepare for my second BIO-International Convention as CEO of ERS Genomics, I’ve taken a moment to reflect on the extraordinary year behind us. CRISPR’s practical impact is accelerating, enabling breakthroughs in treating genetic diseases, engineering more resilient crops, and transforming industrial biotechnology. Every day, researchers use CRISPR to confront some of the world’s most pressing challenges in science, medicine and beyond, reminding us that the future of CRISPR is not tomorrow; it’s already here.
Securing our Position in a Challenging IP Landscape
The CRISPR/Cas9 intellectual property landscape remains dynamic, but recent developments have only reinforced our leadership position.
At ERS Genomics, we provide global access to the foundational CRISPR/Cas9 IP owned by the CVC Group and based on the Nobel Prize-winning research of Profs Emmanuelle Charpentier and Jennifer Doudna. Our licensees, empowered by the confidence that comes with strong, defensible IP, span diverse fields from pharma and life sciences tools to agriculture and synthetic biology. This demonstrates the broad impact CVC’s technology is having on the world we live in.
Looking first at the United States, the U.S Court of Appeals for the Federal Circuit in Washington, D.C., recently ordered the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (PTAB) to reconsider its 2022 decision that scientists at the Broad Institute invented CRISPR/Cas9 gene editing in eukaryotic cells. This allowed them to hold patents with claims for use in eukaryotic calls alongside the patents also granted to the CVC Group for use of CRISPR/Cas9 in all cell types. The Appeals Court determined that PTAB failed to apply the correct legal standard and required it to re-evaluate the evidence of conception. This ruling is a welcome course correction and brings us closer to Profs Charpentier and Doudna being rightfully recognised as the original inventors of CRISPR/Cas9 gene editing technology for all cell types, including eukaryotic cells, as evidenced by their groundbreaking 2012 Science publication.
In Europe, CVC Group took decisive action in 2024 to voluntarily withdraw their EP’811 and EP’400 patents. This was a strategic response to a preliminary opinion from the European Patent Office (EPO) Board of Appeal that diverged from earlier, more favourable rulings by the EPO Opposition Division and which introduced potential procedural vulnerabilities. That tactical decision was vindicated earlier this year when the European Patent Office granted a new CRISPR/Cas9 patent (EP 4 289 948A) to CVC Group, with broad claims covering guide RNAs and their combination with Cas9. As noted in a recent IAM article, the newly granted patent further strengthens the CVC Group’s position as “one of the most important CRISPR patent-owners in Europe.”
Five CRISPR Breakthroughs Transforming Healthcare
The pace of CRISPR innovation is breathtaking. Here are five recent developments that highlight its transformational potential:
1) Casgevy: The first FDA-approved CRISPR-based therapy, Casgevy (exagamglogene autotemcel), is now changing lives for patients with sickle cell disease and transfusion-dependent β-thalassemia. Unlike traditional treatments focused on symptom relief, it offers a one-time, potentially curative therapy that could give patients the possibility of a normal, transfusion-free life.
2) First-in-Human Personalised CRISPR therapy: In February 2025, at the Children’s Hospital of Philadelphia, Baby KJ Muldoon became the first patient treated with a personalised CRISPR gene editing therapy. The treatment targeted severe carbamoyl phosphate synthetase 1 (CPS1) deficiency which is a rare, life-threatening metabolic disorder. This showcases the real-world application of CRISPR for therapeutic intervention in ultra-rare diseases.
3) Fighting Antibiotic Resistance: Scientists have used CRISPR/Cas9 to neutralise MRSA resistance genes, restoring antibiotic efficacy across multiple drug classes and pointing the way toward a powerful new weapon in the fight against antimicrobial resistance.
4) Next-Gen Delivery Systems: Novel peptide-based nanoparticles (ADGN) have shown exceptional promise, achieving 60% knockout efficiency in vitro and successful systemic delivery to lung tumour cells in vivo, overcoming key barriers in CRISPR/Cas9 RNA delivery for cancer treatment.
5) New Discovery Tools: The PAMPHLET bioinformatics pipeline has revolutionised CRISPR/Cas9 spacer identification, achieving an 18-fold improvement in throughput. This opens new possibilities for discovering and validating new CRISPR systems across diverse applications.
Looking Ahead
The pace and breadth of progress we’ve witnessed over the past year reinforces one simple truth: CRIPR is no longer a promise, it’s a platform that’s unlocking solutions to previously intractable problems.
At ERS Genomics, we remain committed to enabling global access to this foundational CRISPR technology. I look forward to engaging with our partners, licensees, the broader life science community at BIO and beyond. Together, we are unlocking the full potential of CRISPR to transform the world we live in for the better.
John E Milad
CEO
ERS Genomics