Original papers and reviews from the Pharma Now community — across therapeutic areas, modalities, technology and manufacturing.
This systematic review synthesises reported clinical outcomes for bispecific T-cell engager (BiTE) constructs in refractory solid tumours, drawing on 47 trials completed between 2019 and 2025. Overall response rates, immune-related adverse events, and cytokine release syndrome management strategies are analysed alongside dosing regimens. Findings suggest that step-dosing protocols meaningfully reduce grade 3+ CRS incidence without compromising efficacy, and that combination with checkpoint inhibitors remains the most promising near-term development path.
The cold chain requirements of first-generation mRNA vaccines constrained deployment in low- and middle-income settings. This paper evaluates four lipid nanoparticle formulation strategies that have demonstrated stability at 2-8°C for six months or longer, comparing immunogenicity, tolerability, and cost impact. We show that ionisable lipid selection and lyophilised formats each deliver meaningful stability gains, but that combination approaches remain necessary to reach the WHO PQ storage benchmark without compromising booster immunogenicity.
KRAS G12C inhibitors have transformed treatment options for a subset of NSCLC and colorectal cancers, but acquired resistance emerges within a median of 6.5 months. This paper characterises resistance mechanisms observed across 214 post-progression biopsies, identifying convergent activation of RTK signalling, secondary KRAS mutations, and epithelial-mesenchymal transition programmes. We propose a three-tier surveillance framework for early resistance detection and outline combination strategies now entering Phase Ib evaluation.
Commercial manufacture of AAV vector-based gene therapies for ultra-rare indications faces a distinctive economics: patient populations below 500 globally, yet CMC expectations calibrated for commercial-scale biologics. This paper reviews the process intensification, analytical characterisation, and comparability protocols that recent approvals have relied on, and quantifies the cost-of-goods trajectory across three sponsor case studies. We argue that shared manufacturing platforms and adaptive CMC frameworks are essential to sustain therapy access for indications below 100 patients per year.