At a glance: Gene editing techniques are pushing the boundaries of personalised medicine, aiming to move beyond expensive, bespoke interventions toward more scalable platforms. Will the foundational CRIPSPR IP saga remain relevant?

On-market therapies and contested ownership

Gene editing has entered a transformative era. Since CRISPR-Cas9’s debut in 2012, and its Nobel Prize in 2020, the technology has enabled precise DNA cuts to disrupt or modify genes. This breakthrough led to Casgevy® (exagamglogene autotemcel) from Vertex Pharmaceuticals, the first approved CRISPR therapy for sickle cell disease (SCD) and β-thalassemia (TDT). Casgevy gained UK approval in November 2023, followed by the FDA in December 2023 and EU conditional market authorisation in February 2024. It works by editing a patient’s own stem cells ex vivo to restore healthy haemoglobin production.

Such gene therapies may rely on licences to the original CRISPR patents, which remain subject to intense, ongoing disputes. For example, immediately after receiving FDA approval for Casgevy, Vertex Pharmaceuticals entered into a licensing agreement with Editas Medicine (which licenses the original Broad Institute CRISPR patents). Meanwhile, foundational ownership remains contested primarily between CVC Group, ToolGen, Broad Institute/Harvard/MIT and Sigma-Aldrich, keeping freedom to operate and licensing strategies in flux.

Approvals for CRISPR/Cas9-based therapies are expected to become increasingly common and so similar licences could be agreed amid this backdrop of dispute. That said, by the time next-generation products come to market, along with a new wave of associated IP, licences to legacy Cas9 portfolios may be of secondary importance.

Beyond CRISPR-Cas9: Base and Prime Editing

While revolutionary, CRISPR-Cas9 relies on double-strand breaks. This can introduce errors, making it better suited for gene disruption (knockouts) than precise correction (knock-ins or substitutions). To overcome these limitations, researchers developed next-generation tools:

• Base Editing: Introduced in 2016, this technique swaps a single nucleotide for another without cutting both DNA strands. Whilst it can correct point mutations responsible for many genetic disorders, it is limited to C↔T or A↔G mutations. Clinical trials are underway for conditions such as alpha-1 antitrypsin (AAT) deficiency and familial hypercholesterolemia (FH), and base editing has already been used in investigational CAR-T therapies for leukaemia.
• Prime Editing: First described in 2019, prime editing acts as a “search-and-replace” tool for DNA, enabling small insertions, deletions, or substitutions, reliably up to ~40 nucleotides. Prime editing is more complex than base editing. It requires larger machinery, which complicates delivery and efficiency. However, it is also more versatile and can perform all 12 possible nucleotide conversions. In May 2025, Prime Medicine reported the first human treatment using prime editing for chronic granulomatous disease, correcting a two-base deletion and restoring immune function.

Challenges: Cost and Access

Casgevy’s US list price, at roughly $2.2 million per patient, makes it one of the most expensive medicines globally. Manufacturing is complex, requiring bespoke cell harvesting, editing, and reinfusion. Access, therefore, remains limited.  However, the once-and-done potential of these treatments could help reduce healthcare costs in the long term.

Current gene editing therapies mainly target rare diseases. A prominent example occurred in February 2025, when clinicians delivered a personalised CRISPR-based editing therapy to an infant with severe carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a disorder affecting roughly 1 in 1.3 million births. While lifesaving, such bespoke treatments highlight scalability challenges, given that there are thousands of rare diseases affecting hundreds of millions of people worldwide.

Scalability as the next phase

The next phase of gene editing is about scalability. Researchers are now pursuing “disease-agnostic” platforms, entailing standard delivery systems combined with modular editing components, so treatments can be rapidly adapted for a variety of conditions.

In late 2025, David Liu’s lab published a Nature paper on PERT (prime editing‑mediated readthrough of premature termination codons), a single‑agent prime‑editing strategy designed to address nonsense mutations (a class of mutations that halt protein production too early), which are estimated to cause a third of genetic diseases. The PERT approach makes an edit that equips cells with the means to produce full-length functional protein, regardless of the gene carrying the pathogenic variant. In cell and animal models PERT restored protein function across multiple diseases, suggesting a plausible route to “one editor, many indications”.

Base and prime editing technologies presently underpin the vision of scalable gene editing. Together, they can correct nearly all known disease-causing variants, and trials are already expanding beyond rare disorders to common conditions like cardiovascular disease and cancer immunotherapy. If successful in implementing a platform-like approach, gene editing could shift from being a niche intervention to a mainstream therapeutic toolkit, addressing both rare and widespread diseases more efficiently.

The future landscape

Patents will naturally play a critical role in protecting next-generation gene editing technologies and securing investment for their development, as they have done for the foundational methods. The next wave of IP is already coalescing around improved editors, delivery methods, manufacturing know‑how, and computational design/off‑target control. These are the layers that help determine whether platforms can scale across indications, and they will draw in new applicants and new litigation. Indeed, ToolGen has already commenced actions tied to Casgevy in the US in relation to RNP delivery technology.  

A significant number of the foundational Cas9 patents are expected to expire by mid-2030. This means by the time more platform‑style editing takes hold, the early Cas9 rights may still command respect but will unlikely command the field. It will be interesting to see who, if anyone, ultimately comes out on top when the dust settles. Will there be a single dominant company, a stable arrangement among multiple licensors, or a pooled solution that makes the tools broadly accessible on predictable terms?

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