THE NEXT FRONTIER OF INDIAN PHARMA: BIOLOGICS AND ADVANCED THERAPEUTICS

Translational research in drug development: challenges and opportunities for pharmacists

Translational Research (TR) constitutes a multidirectional, iterative paradigm designed to facilitate the seamless transition of fundamental biomolecular discoveries into robust clinical interventions. The review critically examines the five - tier (T0 - T4) t ranslational trajectory, identifying the systemic biological and operational bottlenecks - most notably the “Valley of Death” and xenogeneic discordance - that perpetuate the high rates of therapeutic attrition within the pharmaceutical pipeline. Furthermore , it delineates the emergence of the pharmacist as a translational catalyst, uniquely equipped to bridge the gap between abstract molecular hypotheses and patient - centric outcomes through the application of pharmacometrics, Physiologically Based Pharmacoki netic (PBPK) modelling, and Model - Informed Drug Development (MIDD). By integrating multi - modal “omics” data with real - world evidence (RWE), the modern pharmacist optimizes the therapeutic index and ensures the clinical scalability of novel chemical entitie s. This paper posits that a paradigm shift toward Open Science frameworks, systems pharmacology, and precision individualization is imperative for overcoming the epistemological opacity of modern drug discovery and achieving global therapeutic equity.

Industry insights: Clinical advances and regulatory momentum across nucleic acid therapeutic modalities

May and June 2026 saw significant clinical and regulatory momentum across nucleic acid therapeutic modalities and strategic activity included a multi-target ASO collaboration between Servier and n-Lorem Foundation in rare neurodevelopmental disorders, and WuXi AppTec’s announcement of a 17% increase in capital expenditure to expand oligonucleotide and peptide manufacturing capacity globally.

[Drug Discovery - A surge in innovation driven by technology and artificial intelligence].

Engineering the future of advanced therapy medicinal products: a bioengineering call to action

Advanced Therapy Medicinal Products — cell therapies, gene therapies, and tissue-engineered products — are beginning to deliver on the promise of curative medicine: CAR-T therapies double survival in chemotherapy-refractory lymphomas, gene therapies reverse the natural history of spinal muscular atrophy and hemoglobinopathies, and Pluripotent Stem Cell (PSC)-derived islet transplantation renders type 1 diabetic patients insulin-independent. Yet the trajectory from proof-of-concept to equitable, scalable deployment is consistently impeded not only by unresolved biology but also by engineering, manufacturing, logistical, regulatory, and economic bottlenecks that the bioengineering community has not engaged with at the required scale. In this Perspective, grounded in clinical experience across hematological malignancies, monogenic diseases, and metabolic disorders, we identify five rate-limiting bottlenecks where bioengineering intervention is urgently needed and uniquely tractable: scalable and adaptive biomanufacturing; real-time in-process quality control; precise targeted delivery; biomaterial and scaffold engineering for cellular engraftment and immune protection; and data-driven patient stratification constrained by health equity. We argue that the evolving regulatory landscape in Europe — including the European Biotech Act framework and ICH Quality by Design principles — creates structural incentives for engineering-led solutions, and that economic sustainability requires bioengineering to drive down production costs and enable the off-the-shelf transition. We call on the bioengineering community to engage with ATMP translation not as technical support to clinical medicine, but as a constitutive partner shaping its pace, cost, and equity.

The Changing Landscape of Global Biopharmaceutical Leadership, 2004-2024.

For several decades, the United States (U.S.) has been the leader in biopharmaceutical advancements. Recently, the U.S. has increased government interventions to limit prices for patent-protected medicines. While it is unclear whether ongoing efforts to implement international reference pricing in the U.S. could have similar effects, descriptive measures of global biopharmaceutical market dynamics can help benchmark future assessments. In this analysis, we analyzed trends in the geographic distribution of select measures of biopharmaceutical leadership from 2004 through 2024. Using the PharmaProjects database from Citeline, we found that 43.1% of first launches of small-molecule and biologic drugs occurred in the U.S., 15.3% occurred in Europe, and 9.9% occurred in China. Over this time, the share of drugs launched first in the U.S. went from 45.9 to 37.8%, the share of drugs launched first in Europe fell from 26.7 to 8.5%, while this share in China grew from 3.3 to 28.7%. The share of first-launched therapeutics developed by U.S.-headquartered companies and by companies based in Europe declined while the share developed by companies based in China increased. Though additional research is needed on the measurement of biopharmaceutical leadership, and impacts of government intervention, this descriptive study suggests that over several decades, there has been a strong decline in European measures of leadership. At the same time, China is becoming increasingly important in the global biopharmaceutical space. This analysis supports the view that the U.S remains a leader, and offers a benchmark and context for future evaluations that consider potential unintended consequences of ongoing proposals to increase government intervention in the prices of patent-protected medicines.

Biologic prodrugs: inactive protein and peptide drugs and drug conjugates with targeted activation

Biologics have revolutionized the treatment of many diseases, particularly rare and genetic diseases, for which small-molecule drugs have shown limited efficacy, resulting in a surge in regulatory approvals over the past few decades. Despite these advances, biologic therapeutics face significant challenges, including complex formulation, manufacturing constraints, and safety and efficacy concerns following their administration. Issues with poor tissue penetration, reduced absorption across biological barriers, environmental and enzymatic degradation, and rapid clearance challenge efficacy, particularly at the target site, thereby increasing off-target exposure and the risk of adverse events. Biologic prodrugs offer a promising strategy to address these limitations by rendering biologics pharmacologically inactive until selectively activated in response to a defined physiological or pathological cue to confer bioactivity at the target site. This review discusses several biologic prodrugs and their activation mechanisms, including enzymatic and environmental triggers, such as pH changes, hypoxia, redox potential, and external stimuli (e.g. light). We explore various biologic prodrugs, including protein and recombinant fusion proteins, antibody-drug conjugates (ADCs), polymer-drug conjugates, and cell-penetrating peptides (CPPs) as masking motifs, as well as enzyme-prodrug therapy (EPT) systems, i.e. gene-directed enzyme prodrug therapy (GDEPT) and antibody-directed enzyme prodrug therapy (ADEPT). We also discuss lysosome-targeted biologic prodrugs that leverage the acidic, enzyme-rich, and compartmentalized lysosomal environment for controlled activation. Collectively, the examples discussed show biologic prodrugs as a versatile platform that selectively responds to physiological and environmental triggers, enabling enhanced spatial and temporal control of drug activation and thereby limiting systemic toxicity and other challenges associated with biologics.