29 TWENTYFOURSEVENBIOPHARMA Issue 2 / June 2025 promise in matching patients to clinical trials, with early findings suggesting faster recruitment and reduced administrative burden. AI’s capabilities also extend towards ensuring a more dynamic and patient-focused clinical trial design. This is achieved by harnessing the AI algorithm to analyse real-world data (RWD) and identifying the patient subgroups more likely to respond positively to treatments. This enables real-time adjustments based on patient responses for better outcomes. The financial proposition is compelling. Coherent Solutions predicts that with AI’s integration “pharma companies can expect up to $25 billion (€22.06bn) in savings in clinical development alone.” Perhaps with one eye on the future, generative AI has caused a few waves among industry observers, who point out its ability to generate entirely new data, such as images, sentences, audios, videos, and novel chemical molecules. Keen to prove its mettle in drug discovery, Roche recently revealed details of its “lab in a loop” mechanism, where lab and clinical data are used to train AI models and algorithms designed by the pharma company’s researchers. From there, the trained models are used to make predictions on drug targets, therapeutic molecules. These predictions are further investigated in the lab, generating new data to further tweak existing models. This iterative process streamlines the traditional trial-and-error approach, leading to faster and more effective therapies. “To take advantage of these new approaches and to apply them rapidly, we need to bring together expertise from different disciplines - by doing so we have a tremendous opportunity to hopefully bring medicines to patients faster than we do today,” commented John Marioni, Senior Vice President and Head of Computational Sciences at Genentech, a member of the Roche Group. Advancing cell and gene therapy As AI advances drug discovery and development, adjacent fields are also benefiting. The rapidly evolving landscape of cell and gene therapy is a prime example, where precision tools and datadriven insights are driving breakthroughs once thought impossible. In the US, Neurotech’s Encelto, an intraocular implant of encapsulated engineered human cells, was recently approved for the treatment of macular telangiectasia type 2 (MacTel), as reported by the American Society of Gene & Cell Therapy (ASGCT) in their Q1 2025 report. Additionally, the approval of Alnylam’s siRNA therapy, Qfitlia, for the treatment of haemophilia A and B marked a pivotal moment for RNA interference (RNAi)-based therapeutics, highlighting the growing clinical relevance of gene-silencing strategies in managing complex, inherited disorders. In China, Platinum Life’s Ruiboshen received approval for the country’s first mesenchymal stem cell therapy for steroid-refractory acute graftversus-host disease. The American Society of Gene and Cell Therapy (ASGCT) report a continuing trend of increased clinical trial numbers for non-genetically modified cell therapies in non-oncology indications - up to 74% in Q1 2025 compared to 58% from the previous quarter. Conversely, gene therapy trials are shifting towards oncology. Of the 79 trials initiated in Q1, 57% were for oncology indications, the highest oncology trial proportion for the past year. But approvals aside, the field continues to push boundaries, especially in gene editing, where the CRISPR-Cas9, remains a revolutionary tool. The UK MHRA’s approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel (CASGEVY) in November 2023 marked a pivotal moment, being the first marketing authorization granted to a CRISPR gene editing therapy. The success of mRNA technology, initially driven by COVID-19, is proving useful beyond infectious diseases by proving its worth in cancer immunotherapy, genetic disorders, and regenerative medicine. Companies like Moderna and BioNTech, global leaders in mRNA innovation, are expanding their pipelines to include vaccines for RSV, CMV, seasonal influenza, and personalized cancer treatments, reinforced by partnerships. Navigating biotech’s investment landscape This wave of innovation, from mRNA to gene and cell therapies, is naturally drawing the attention from investors seeking to capitalize on biotech’s next breakthroughs. As scientific milestones accumulate, the investment landscape too must evolve. Investment in biotech remains strong but more selective, with late-stage assets (phase 2 and beyond) receiving larger deals. J.P. Morgan and BIOTECH
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