The CRISPR therapeutics landscape in 2025

The CRISPR therapeutics landscape in 2025

Once confined to science fiction, precise genetic engineering has become a reality and has profoundly impacted modern medicine.
 
CRISPR technology, leveraging guide RNA (gRNA) base pairing to modify target sequences, originates from prokaryotic immune systems, using a Cas enzyme to cleave DNA at specific sites1,2. Capable of targeting both DNA and RNA, CRISPR has driven remarkable advancements, earning its pioneers a Nobel Prize3. Since the breakthrough discovery of CRISPR as a tool for genetic engineering in 2012, CRISPR-based therapeutics have progressed from experimental research to licensed treatments. Multiple significant milestones have already been reached in 2025. In January, NICE in the UK approved Casgevy® for severe sickle cell disease4,5. Even more recently in May, researchers reported the world’s first successful patient-specific in vivo gene editing treatment for severe carbamoyl-phosphate synthetase 1 (CPS1) deficiency6.
 
In this article we provide a snapshot of some of the more recent patent and scientific developments in the field of CRISPR-based therapeutics.
 

European patent developments

The high-value therapeutics market has led to extensive patenting of CRISPR systems and methods. The ongoing patent battle among early CRISPR pioneers saw the University of California withdraw its own European patents EP2800811 and EP3401400 after an unfavourable opinion from the Board of Appeal7. By withdrawing the patents before any potential revocation, the patentee avoids having the unfavourable finding from being recorded as the reason for revocation, which could further impact any related patents.
 
Meanwhile, Editas Medicine (a licensee of the Broad Institute’s CRISPR patents) reached a licensing agreement with Vertex Pharmaceuticals, developers of Casgevy®. The agreement involved an upfront $50 million fee and annual payments between $10 million and $40 million until 2034, when the relevant patents expire8.
 
In a recent development, the gene editing company ToolGen has sued Vertex, Lonza and Roslin Cell Therapies in the UK Patents Court for alleged infringement of its CRISPR-Cas9 patent EP4357457 in relation to Casgevy®9. Notably, ToolGen has stated that the lawsuit is not aimed at restricting patient access to Casgevy® but they are seeking compensation through a licencing agreement for the alleged use of ToolGen’s CRISPR technology. The infringement action follows multiple oppositions filed against ToolGen’s patent EP4357457 and revocation actions by Vertex in the Netherlands.
 
As with any emerging technology, the intersection of research, investment, and intellectual property rights continues to shape the field. The ToolGen Vertex litigation will be particularly interesting to watch in the coming months, being the first patent infringement litigation relating to CRISPR in the UK.
 

Recent advancements in CRISPR technology

In 2025, we have already seen numerous reports of exciting breakthroughs utilising CRISPR technology worldwide. Provided below is a selection of some interesting examples from research institutes and biotechnology companies alike.
 
As mentioned above, teams at Children’s Hospital of Philadelphia and Penn Medicine have successfully treated a rare genetic disorder in an infant, using a customised CRISPR base editing therapy6. Following the patient’s diagnosis of CPS1 deficiency, within six months the researchers developed a personalised liver-targeted base editing therapy delivered via lipid nanoparticles to correct the defective CPS1 enzyme. The therapy functions through the Cas enzyme directing a deaminase to the target residue, which is then converted to the correct base (adenine to guanine). After administration of the therapy, the researchers were able to reduce the patient’s medication and increase their dietary protein, without adverse side effects, indicating successful treatment. While monitoring for long-term efficacy of the treatment is ongoing, this breakthrough may precipitate rapid deployment of patient-specific gene-editing therapies for many genetic diseases.
 
A Chinese Academy of Sciences study recently showcased the power of CRISPR by creating mice with two biological fathers10. Researchers modified 20 key imprinted loci, affecting hundreds of genes, enabling embryos that would normally be unviable to develop into fully mature animals. These results have implications for regenerative medicine and complex genetic engineering.
 
UK researchers have developed CRISPR MiRAGE (miRNA-activated genome editing), a technique allowing tissue-specific gene editing by leveraging miRNA signatures11. Successfully tested in Duchenne muscular dystrophy mouse models, this method enhances cell specificity and minimises off-target effects.
 
Intellia Therapeutics has initiated a Phase 3 trial of NTLA-2002, a CRISPR-Cas therapy targeting hereditary angioedema (HAE)12. By inactivating the KLKB1 gene, NTLA-2002 aims to prevent HAE attacks, with earlier trials showing promise after a single dose. If successful, this therapy could become the first one-time treatment for HAE, potentially launching in the U.S. by 2027. The therapy has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the U.S. FDA, PRIME Designation from the EMA, and an Innovation Passport from the UK MHRA.
 
In cancer therapy, CRISPR-engineered chimeric antigen receptor natural killer (CAR-NK) cells have been developed by integrating CAR sequences into the GAPDH 3'UTR locus of NK-92MI cells13. This site-specific integration enhances receptor expression, improves anti-tumour activity, and reduces metabolic reliance compared to lentiviral CAR-NKs. These findings present a promising new strategy for cancer immunotherapy and further refinement of CAR-NK cells using CRISPR technology.
 

CRISPR delivery systems

The success of CRISPR-based therapies depends not only on the gene-editing mechanism but also on efficient delivery systems. While CRISPR enables precise gene targeting with minimal off-target effects, delivering the CRISPR-Cas9 machinery in vivo remains a challenge, necessitating robust delivery platforms14. These include adeno-associated viruses (AAVs), lentiviruses, lipid nanoparticles (LNPs), polymer nanoparticles, peptide-based nanoparticles, and inorganic nanoparticles, which are further reviewed here.
 
A notable recent advancement in LNP technology is the development of biodegradable ionizable lipids using the Passerini reaction, enabling mRNA delivery. Researchers at the University of Toronto identified an LNP-formulated ionizable lipid (denoted A4B4-S3) that outperforms the clinical benchmark lipid (SM-102) in delivery of mRNA to the liver in mice15. SM-102 was a key component of Moderna’s highly successful COVID-19 vaccine, suggesting that further improvements in LNP technology will benefit CRISPR therapeutics16.
 
Patent activity in lipid nanoparticle-based RNA delivery has surged, with numerous filings protecting novel cationic lipids essential for transfection efficiency and safety17. Recent research found that, of patents relating to LNPs that deliver siRNA, the percentage of patents specifically claiming cationic lipid structure has risen from 9% in 2003 to 50% in 202117. Beyond developing and patenting new lipids, researchers are exploring optimal formulation ratios and novel mechanisms for targeted delivery such as receptor-targeted RNA delivery18, demonstrating the vital importance of delivery systems in RNA-based and CRISPR therapeutics19.
 

Conclusion

CRISPR continues to provide fertile ground for scientific and medical advancements in 2025. From approved treatments for genetic disorders to groundbreaking research in tissue-specific editing and cancer immunotherapy, the field continues to push biotechnology’s boundaries. Delivery mechanisms, particularly LNPs and other nanoparticle-based systems, are evolving to address the challenges of precise therapeutic administration. With research, investment, and intellectual property shaping this dynamic landscape, CRISPR technology still holds the potential to revolutionize medicine, providing solutions for previously untreatable conditions. While the journey from discovery to clinical application continues, CRISPR remains a driving force in the future of genetic medicine.
 
Our team has considerable experience advising clients on patent matters in relation to CRISPR-based genome editing and technologies such as mRNA vaccines, RNA interference (RNAi), antisense oligonucleotides (ASO), non-coding RNA, and LNP chemistry. Our experts include Beth Ormrod, Jake Rightmyer, Jamie Atkins, Jessica Duncombe, Juliette Howarth, Nick Lee, Oliver Lam and Samuel Bailey. Please get in touch if you have any related queries! 
 


1. Jinek M., et al., A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012 Aug 17;337(6096):816-21. doi: 10.1126/science.1225829. Epub 2012 Jun 28.

2. Ran, F., et al. Genome engineering using the CRISPR-Cas9 system. Nat Protoc 8, 2281–2308 (2013). https://doi.org/10.1038/nprot.2013.143

3. https://www.nobelprize.org/prizes/chemistry/2020/press-release/

4. https://www.nice.org.uk/news/articles/nice-approves-groundbreaking-one-off-gene-therapy-for-severe-sickle-cell-disease

5. https://www.kilburnstrode.com/knowledge/european-ip/world-s-first-crispr-based-therapy-offered-nhs-in

6. Musunuru K., et al., Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. N Engl J Med. 2025 May 15. doi: 10.1056/NEJMoa2504747. Epub ahead of print.

7. https://www.technologyreview.com/2024/09/25/1104475/nobel-prize-winners-cancel-crispr-patents-europe/

8. https://www.technologyreview.com/2023/12/13/1085209/vertex-license-controversial-crispr-patent-editas/

9. https://caseboard.io/cases/fcb498bf-885f-40ff-805f-44e1545b8fe3

10. Li Z. K., et al., Adult bi-paternal offspring generated through direct modification of imprinted genes in mammals. Cell Stem Cell. 2025 Mar 6;32(3):361-374.e6. doi: 10.1016/j.stem.2025.01.005. Epub 2025 Jan 28.

11. Garcia-Guerra A., et al., Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs, Nucleic Acids Research, Volume 53, Issue 2, 27 January 2025, gkaf016, https://doi.org/10.1093/nar/gkaf016

12. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-first-patient-dosed-haelo-phase

13. Dai L., et al., CRISPR knock-in of a chimeric antigen receptor into GAPDH 3’UTR locus generates potent B7H3-specific NK-92MI cells. Cancer Gene Ther 32, 227–239 (2025). https://doi.org/10.1038/s41417-025-00872-1

14. Sioson V. A., et al., Challenges in delivery systems for CRISPR-based genome editing and opportunities of nanomedicine. Biomed Eng Lett. 2021 Jul 13;11(3):217-233. doi: 10.1007/s13534-021-00199-4.

15. Xu Y., et al., Rational design and modular synthesis of biodegradable ionizable lipids via the Passerini reaction for mRNA delivery, Proc. Natl. Acad. Sci. U.S.A. 122 (5) e2409572122 (2025), https://doi.org/10.1073/pnas.2409572122.

16. Zhang, L., et al. Effect of mRNA-LNP components of two globally-marketed COVID-19 vaccines on efficacy and stability. npj Vaccines 8, 156 (2023). https://doi.org/10.1038/s41541-023-00751-6

17. Yunfeng Han Y., et al., Profiling patent compounds in lipid nanoparticle formulations of siRNA, Molecular Therapy - Nucleic Acids, Volume 35, Issue 4, 2024, 102362, ISSN 2162-2531, https://doi.org/10.1016/j.omtn.2024.102362.

18. Witten J., et al., Recent advances in nanoparticulate RNA delivery systems, Proc. Natl. Acad. Sci. U.S.A. 121 (11) e2307798120 (2024), https://doi.org/10.1073/pnas.2307798120.

19. Tenchov R., et al., Lipid Nanoparticles─From Liposomes to mRNA Vaccine Delivery, a Landscape of Research Diversity and Advancement. ACS Nano. 2021 Nov 23;15(11):16982-17015. doi: 10.1021/acsnano.1c04996. Epub 2021 Jun 28.

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