One Nucleus convened this year’s ON Helix event at the Babraham Research Campus in Cambridge, attended by Bristows’ Jamie May and Erik Müürsepp, under the banner of “Disrupting Bio Innovation”. The conference explored the key trends currently driving change in the life sciences sector, with a particular focus on the breakthrough technologies, policies and business models that are shaping the therapeutic discovery and development landscape – which we set out to summarise in this article. At its core, the business of drug discovery and development remains challenging. Carrying a new medicine through the process of discovery all the way through to regulatory approval and commercial launch generally still takes well over a decade and is extremely capital-intensive. Furthermore, only a very small proportion of candidates achieve final approval, even after reaching the clinical testing stage, with many ultimately failing (often due to toxicity or lack of efficacy). The new tools, treatments and methodologies explored at ON Helix – the “disruption” of bio innovation alluded to in the framing of the conference – are best understood as a response to this backdrop: an effort to make the discovery and development process faster, cheaper and more effective.
1. An AI-led revolution in drug discovery and development?
Unsurprisingly, artificial intelligence was a major topic of discussion. The clear consensus is that AI has begun to revolutionise the drug discovery process. Following traditional methods, it might take up to four years to reach the point of development candidate nomination. But that timeline is being significantly compressed via the use of computational platforms and AI, notably in target identification (through the analysis of vast biological datasets) and molecule design (using generative chemistry and prediction models).
Insilico Medicine, which applies its generative “Pharma.AI” platform across the discovery-to-clinic pipeline, offers a striking illustration. The Hong Kong-listed and Boston-headquartered biotech’s lead candidate, rentosertib – an oral small-molecule inhibitor being developed to treat idiopathic pulmonary fibrosis (a progressive lung disease with high unmet medical need) – went from initial target identification to preclinical candidate nomination in around 18 months, and has very recently progressed into Phase III trials. As well as developing its own pipeline of candidates (a number of which have reached clinical stage), Insilico has struck lucrative deals with a number of major pharmaceutical companies keen to access its discovery platform. In 2026 alone, Insilico announced R&D collaborations with Eli Lilly (worth up to US$2.75 billion), SK Biopharmaceuticals (up to US$2.5 billion) and Takeda (up to US$600 million), on top of earlier deals with the likes of Sanofi and Servier. Meanwhile, in the UK, Isomorphic Labs (the AI-first drug discovery company spun out from Google DeepMind in 2021) continues its remarkable rise, having signed collaborations with Eli Lilly and Novartis (in 2024) and raised substantial external capital (US$2.1 billion in 2026) to develop its own pipeline. Ultimately, this scale of investment, and the readiness of established pharma companies to entrust AI-led platforms with the early stages of drug discovery and development, reflect a growing confidence within the industry that these tools are capable of delivering valuable results.
As a number of speakers stressed, however, a note of caution is warranted here. For all the headlines and capital invested, AI has so far been predominantly deployed at the front of the pipeline (i.e. in target identification, molecule generation and lead optimisation), where hypotheses can be explored cheaply and iteratively in silico. Its impact on the later, more expensive stages of development and testing has, to date, been far more limited, and it remains to be seen whether AI platforms can successfully predict how a molecule will interact with the complexity of the human body, and whether candidates can demonstrate safety and efficacy in late-stage trials (where most drugs fail). Indeed, Insilico’s rentosertib currently stands alone as the first drug with both an AI-discovered target and an AI-designed molecule to reach late-stage trials. For now, then, AI is best understood as compressing the earlier stages of discovery and reducing the time from lab to clinic rather than transforming drug development as a whole. The ultimate test – success in the clinic and market approval – still lies ahead.
2. Key therapeutic modalities and the pivot from ex vivo to in vivo
If the topic of AI dominated discussions around how treatments are discovered and developed, cell and gene therapy was the standout theme in conversations around what exactly is being developed.
One promising trend identified was the evolution of CAR-T cell immunotherapy from its conventional form – a bespoke ex vivo process involving the extraction of a patient’s own T-cells and their re-engineering in the lab before being re-infused into the body – towards in vivo approaches that reprogram immune cells inside the patient via intravenous injection. The ex vivo model, while effective in treating blood cancers such as leukaemia and lymphoma, has presented challenges due to its cost, potential serious adverse effects and the complexity of delivery. The in vivo model, by contrast, promises something closer to a scalable, off-the-shelf medicine – though the treatment remains in early clinical development and more clinical data will be required to prove its safety and effectiveness.
A further area of excitement is the expansion of cell therapy’s reach beyond oncology and into autoimmune disease. Many autoimmune conditions, lupus among them, are driven by rogue B cells that turn the immune system against the body’s own healthy tissue. CAR-T therapy can be used to hunt down and clear out these rogue cells, in effect “resetting” the immune system rather than merely suppressing it, as conventional drugs do – which has the potential to achieve enduring, even drug-free, remissions. British biotech Autolus (a 2014 UCLB spin-out on which Bristows advised), for instance, has reported encouraging early results treating severe, treatment-resistant lupus with its ex vivo CAR-T therapy, with signs of exactly this kind of immune reset. There are also indications that it should be possible to achieve the same effect using an in vivo method, which should ultimately make these therapies cheaper and more widely available. This is no longer purely theoretical: a number of patients with refractory lupus have now been treated with CAR-T cells generated inside the body, with promising early results – offering hope that this could prove an effective therapeutic strategy for autoimmune diseases.
A central question running through these discussions is how best to deliver such in vivo therapies – that is, how to get the reprogramming instructions into the right cells inside the body. Two approaches are now widely established for this purpose. The first uses viral vectors: engineered, harmless viruses (such as lentiviruses) that carry the genetic instructions into the patient’s cells, producing a long-lasting effect. The second packages the instructions as mRNA within tiny lipid nanoparticles – the same form of delivery technology that powered the COVID-19 vaccines – producing only a temporary effect, but one that is more controllable and easier to manufacture at scale. The likely outcome, speakers suggested, is not that one approach prevails outright, but that the choice of delivery vehicle will vary between indications, depending in particular on whether a lasting or a short-lived effect is desired.
This delivery challenge helps to explain one of the more striking commercial dynamics on display: rather than waiting to acquire proven, finished drugs, major pharmaceutical companies are increasingly racing to buy up the underlying platforms – namely, the delivery and cell-engineering technologies – that make in vivo therapies possible, and are doing so far earlier than has traditionally been the case. The bet, in effect, is on the underlying technology rather than on any single product. The result has been something close to a dealmaking frenzy in the in vivo space. In 2025, AstraZeneca acquired the Belgian biotech EsoBiotec and its in vivo cell-engineering platform for up to US$1 billion, and AbbVie paid US$2.1 billion for Capstan Therapeutics and its lipid-nanoparticle platform – despite its lead candidate sitting at an early stage of clinical development. Eli Lilly has been the most acquisitive of all: in the space of barely two months in early 2026 it snapped up two in vivo CAR-T specialists, acquiring Orna Therapeutics in a deal worth up to US$2.4 billion and then Kelonia Therapeutics for up to US$7 billion. Big pharma’s appetite for acquiring early-stage platforms sits within the context of a broader surge in dealmaking driven by the approaching patent cliff. As a wave of blockbuster drugs faces loss of exclusivity, companies are under pressure to secure the technologies they believe will underpin the next generation of medicines – a theme Bristows has explored in Off the cliff, into the deal room.
3. Beyond cell therapy – a broadening toolkit
While cell and gene therapies were most prominently discussed, speakers were keen to stress that these represent just one part of a broadening therapeutic toolkit. Antibody-drug conjugates (“ADCs”) – which attach a chemotherapy drug to an antibody that delivers it directly to diseased cells, sparing healthy tissue – continue to advance, as do novel small-molecule approaches such as targeted protein degradation. Interest in peptide therapies, meanwhile, has been boosted by the success of GLP-1 receptor agonists such as semaglutide in promoting weight loss. Once a fairly niche area, the widespread adoption of these drugs has moved the conversation on from whether they work and towards refining the quality of the weight loss they produce, developing oral formulations, and exploring their potential across a widening range of indications – from cardiovascular disease to addiction.
Some of the most striking data, however, concerned prevention rather than treatment. Speakers pointed to the remarkable impact of the HPV vaccination programme, with recent UK data showing that cervical cancer has been almost eliminated among young vaccinated women. In a similar vein, the monoclonal antibody therapy teplizumab (also known as Tzield) has been recommended by NICE and approved in the US as the first therapy capable of delaying the onset of type 1 diabetes – a shift from managing a disease to intercepting it before it takes hold.
4. Rethinking the preclinical model – the retreat from animal testing
A further theme, explored in sessions focusing on the preclinical and regulatory landscape, was the growing effort to reduce reliance on animal testing. For decades, animal studies have been a mandatory part of the development process leading to human trials, yet they are often imperfect predictors of how a drug will behave in humans. Attention is increasingly turning to so-called New Approach Methodologies (“NAMs”): human-relevant alternatives to animal models. A particular focus of discussion was the growing role of Complex In Vitro Models (“CIVMs”) – laboratory systems engineered to reproduce the structure and behaviour of real human tissue. By way of example, Cambridge-based CN Bio presented on its PhysioMimix platform, an “organ-on-a-chip" device that uses living human cells to mimic the functioning of a real organ. A contrasting approach attracting growing interest and investment is the computational generation of "digital twins" – virtual models that simulate human biological responses in silico – to predict how a patient or tissue might respond without any physical experiment at all.
This is an area in which the regulatory ground is shifting. Regulators in the US, the EU and the UK are being urged to “move at Chinese speeds” as evidenced by this recent call from EFPIA. The EU has committed to strengthen the competitiveness of the EU’s health biotechnology sector through the Biotech Act, which is designed to promote innovation and streamline the regulatory frameworks. Similarly, in April 2025, the US Food and Drug Administration announced plans to begin phasing out its animal-testing requirements for certain drugs, with the MHRA in the UK moving in a similar direction.
Speakers were clear, though, that regulators will embrace these tools only as fast as the evidence allows: confidence has to be earned, and the bar for toxicology in particular will remain high. The direction of travel is nonetheless evident – and as human-relevant models mature from novel techniques into routine infrastructure, they are likely to become an area of significant commercial investment in their own right.
5. A shifting ecosystem
Finally, several speakers reflected on how the structure of the industry itself is gradually shifting. Where the early-stage innovation that fills the pipeline was once largely the preserve of big pharma, today it is emerging biopharma companies that originate the majority of new clinical candidates. That shift helps to explain the dealmaking dynamics described above. The continuing trend is of the largest pharmaceutical companies, facing the loss of exclusivity on a raft of blockbuster products, looking outward – to partnerships, collaborations and acquisitions – to replenish their pipelines. China recurred throughout these discussions, cast both as a formidable competitor and as an increasingly important source of innovation and licensing opportunities – a topic which Bristows has covered in depth.
Concluding remarks
Taken together, the discussions at ON Helix painted a picture of a sector in which innovation is driving meaningful change: the tools of discovery are becoming faster and more capable, the range of therapeutic modalities is broadening, and long-established models of testing and development are being rethought. The consistent note of realism running through the day, however, was that much of the progress to date remains concentrated at the earlier stages of the discovery and development process. The later stages – clinical trials, regulatory approval, manufacturing at scale and, ultimately, pricing and patient access – remain as challenging as ever, and it is here that the "disruption" occurring upstream has yet to prove its worth. The same logic seems to be reflected in recent dealmaking activity. Increasingly, value is being placed on assets at the earlier stages of the development pipeline – on the platforms and enabling technologies that underpin drug discovery and have the potential to generate a whole pipeline of candidates – as opposed to individual candidates. And, as ever, the science will only translate into long-term value if the commercial and legal foundations are sound – from securing and protecting intellectual property in platforms and datasets, to structuring the collaborations, licences and acquisitions through which innovative technologies change hands. These are themes we will be following with interest in the year ahead.

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