Drug & vaccine development

Drug and vaccine development is the multi-stage pipeline — target identification, hit-to-lead screening, lead optimization, preclinical testing, then Phase I-III clinical trials and regulatory submission — that converts a validated biological target into an approved medicine, at a cost of $0.5-3.5 billion and 7-15 years per product with a success probability of just 10-30% depending on product type. Moderna and Merck’s personalized mRNA cancer vaccine, intismeran autogene (formerly mRNA-4157/V940), encodes up to 34 patient-specific tumor neoantigens delivered via lipid nanoparticles; June 2026 data showed a 49% lower melanoma recurrence risk when combined with checkpoint inhibitor therapy, positioning it for regulatory submission. BioNTech, having pivoted its mRNA platform from infectious disease toward oncology, presented late-stage pipeline data at the 2026 ASCO and ELCC conferences for candidates including pumitamig and gotistobart, alongside multiple lung-cancer trial-in-progress programs. In China, CanSino Biologics continues advancing its proprietary mRNA vaccine platform, reporting Phase II safety and immunogenicity data for its CS-2034 mRNA vaccine in Chinese adults. Eli Lilly has parlayed its GLP-1 dual-agonist tirzepatide (Mounjaro/Zepbound) into a portfolio strategy spanning diabetes, obesity and expanded cardiometabolic indications, reinforcing its position at the January 2026 JPMorgan Healthcare Conference even amid looming patent challenges. The regulatory landscape is itself shifting fast: the EU’s 2023-2024 pharmaceutical legislation reform proposes cutting standard data-exclusivity periods from 8 to 6 years while offering the 2 years back only to companies launching simultaneously across all 27 member states, a plan the EFPIA industry federation has sharply criticized as weakening European competitiveness against the US; China’s 2015 IND reform shifted from a permission-based to a notification-based system, driving a fivefold increase in clinical trials and pushing the country’s active trial count (4,700+) to second globally behind the US (12,000+).

The key directions of drug and vaccine development are:

1. Target identification and hit-to-lead screening: AI-assisted molecular library screening to identify and validate biological targets, then discover initial lead compounds.
2. Phase-based clinical development: sequential Phase I (20-100 volunteers, safety), Phase II (100-500 patients, efficacy) and Phase III (1,000-5,000 patients, pivotal) trials building the evidence base for regulatory approval.
3. Personalized mRNA vaccine platforms: patient-specific neoantigen-encoding mRNA vaccines manufactured individually from each patient’s tumor biopsy, extending mRNA technology from infectious disease into oncology.
4. Regulatory-acceleration pathways: FDA Breakthrough Therapy/Fast Track/RTOR, EMA’s proposed exclusivity reform, and NMPA’s Priority Review (cutting review time from 180 to 60 days) all competing to shorten time-to-market for qualifying candidates.

Sectoral value chain#

[Target identification & validation] ──> [Hit-to-lead screening (AI-assisted)] ──> [Lead optimization]
                                                                                              │
                                                                                 (Preclinical in vitro/in vivo testing)
                                                                                              │
     [Approval & market launch → Phase IV surveillance] <──── [Regulatory submission (NDA/BLA/MA)] <─── [Phase I → II → III trials]

Value chain levels#

Cross-cutting technologies of the sector:

• AI-assisted target and molecule design: generative design algorithms applied to both small-molecule drug candidates and mRNA vaccine sequence/neoantigen selection, compressing early-stage discovery timelines.
• Personalized mRNA cancer vaccine manufacturing: synthesizing an individual mRNA vaccine encoding a specific patient’s tumor neoantigens from biopsy sequencing data, requiring per-patient rather than per-batch production logistics.
• Lipid nanoparticle (LNP) delivery: the delivery vehicle that made mRNA vaccines and personalized cancer vaccines clinically viable, protecting mRNA payloads through cellular uptake and endosomal escape.

The drug and vaccine development stack combines computational discovery tools with the clinical and regulatory infrastructure needed to prove safety and efficacy at scale:

1. AI-assisted hit-to-lead screening:
   • Generative and predictive machine-learning models screen virtual compound libraries and mRNA sequence/neoantigen combinations far faster than traditional wet-lab screening, compressing the discovery phase that historically consumed years of a drug’s development timeline.
2. Personalized mRNA cancer vaccine manufacturing:
   • Each patient’s tumor biopsy is sequenced to identify tumor-specific neoantigens, which are then encoded into an individually synthesized mRNA vaccine formulated in lipid nanoparticles — a fundamentally different manufacturing logistics model than standard batch-produced pharmaceuticals.
3. Phase-based clinical trial design:
   • The sequential Phase I (safety, 20-100 volunteers), Phase II (efficacy, 100-500 patients) and Phase III (pivotal confirmation, 1,000-5,000 patients) structure remains the core evidentiary framework regulators require, even as accelerated pathways compress timelines between phases for qualifying candidates.