Open-Science Initiatives in Drug Discovery: The Vision of Target 2035

1. Open-Science Drug Discovery: The Target 2035 Vision Transforming pharmaceutical research through global collaboration, shared knowledge, and democratized access to molecular tools for human health

2. The Challenge: Unlocking the Dark Proteome The human genome encodes approximately 20,000 proteins, yet after decades of intensive pharmaceutical research, fewer than 10% have been studied in sufficient detail to understand their biological functions or therapeutic potential. This vast unexplored territory—often called the "dark proteome"—represents both a significant gap in our scientific knowledge and an enormous opportunity for discovering new treatments. 20K Human Proteins Total encoded by genome Target 2035, initiated by the Structural Genomics Consortium (SGC), aims to bridge this divide by developing chemical probes or molecular tools for every human protein by 2035. These small molecules, which modulate specific protein activities, serve as essential research instruments for investigating protein function across diverse biological contexts. <10% Well-Studied Proteins with detailed understanding 2035 Target Year Complete proteome coverage

3. The Power of Open Collaboration Traditional pharmaceutical research has long operated through secrecy, with proprietary data, patents, and exclusive intellectual property rights restricting access to valuable molecular tools. This siloed approach creates redundant efforts across laboratories, delays target validation, and slows the translation of discoveries into therapeutic innovations. Data Transparency Pre-competitive Collaboration No IP Barriers All experimental data, protocols, and results made openly accessible to the global scientific community Shared infrastructure and resources eliminate duplication of effort across institutions Chemical probes freely available without patent restrictions, accelerating research worldwide Target 2035 transforms drug discovery from a linear, proprietary race into a cooperative ecosystem where insights can be rapidly exchanged and built upon. This collective approach enables scientists everywhere to explore understudied proteins and contribute to identifying new drug targets for diseases that currently lack effective therapies.

4. Enhancing Scientific Rigor Through Openness Reproducibility Crisis Solved Open science addresses two critical issues that have plagued biomedical research: reproducibility and robustness. By making experimental data, protocols, and results openly accessible, Target 2035 allows independent verification and cross-laboratory comparison, ensuring discoveries stand on firm foundations. Public datasets enable integration of AI and machine learning tools that thrive on large, diverse data inputs. AI models can mine repositories of protein structures, chemical probe activities, and cellular phenotypes to predict new druggable interactions and design novel molecules with improved efficacy. Shared Data AI Analysis Open repositories fuel discovery Smarter algorithms emerge New Hypotheses Continuous Loop Testing generates insights Self-reinforcing progress

5. Navigating the Funding Challenge Open-science drug discovery faces substantial challenges, particularly concerning funding models and intellectual property. Traditional pharmaceutical research depends on IP protection to justify large R&D investments—patents ensure financial returns by granting exclusive marketing rights. In contrast, Target 2035 operates in the pre-competitive research phase, relying on public grants, philanthropic contributions, and collaborative partnerships. The Sustainability Question Industry Hesitation Risk of Stagnation Generating chemical probes for thousands of proteins requires significant resources for synthesis, characterization, and validation. Sustaining this funding model over decades presents a formidable challenge. Without IP protection, some worry that private-sector participation may be limited in later development stages, where scaling from chemical probe to clinical drug candidate requires massive investment and regulatory expertise. Critics argue that without clear economic incentives, open-science efforts risk losing momentum once initial enthusiasm fades and funding sources become scarce.

6. Emerging Hybrid Models: Balancing Openness and Sustainability The "Coopetition" Framework To balance openness with sustainability, innovative hybrid models are emerging that blend cooperation and competition. These approaches preserve the benefits of open science while creating pathways for commercial development. Companies may contribute data or expertise to Target 2035 in exchange for early access to certain probes or shared credit in publications. Open licensing strategies allow discoveries to remain accessible while commercialization pathways are structured through equitable agreements. 01 02 Public Investment Recognition Strategic Industry Partnership Governments view open biomedical infrastructure as a public good, similar to the Human Genome Project, catalyzing innovation across sectors Private companies engage in pre-competitive phase while maintaining commercial opportunities for downstream development 03 04 Equitable Access Framework Neglected Disease Focus Licensing structures ensure discoveries remain accessible while enabling sustainable commercialization pathways Open frameworks especially valuable where commercial incentives are weak but medical need is high

7. The Transformative Potential of Target 2035 "If successful, Target 2035 will equip scientists with molecular tools for virtually every human protein, unlocking new understanding of biology and disease at an unprecedented scale." Faster Discovery Democratized Access Novel Therapies Early-stage drug discovery becomes more rapid, efficient, and cost-effective through shared resources Scientists worldwide can explore the dark proteome regardless of institutional resources Earlier identification of therapeutic targets and biomarkers leads to treatments for currently untreatable conditions

8. A Collective Pursuit for Human Health Open science fosters a culture of trust, transparency, and shared purpose—qualities essential for tackling global health challenges that transcend national and corporate boundaries. In an era where scientific collaboration has never been more critical, Target 2035 stands as a testament to what humanity can achieve when discovery is treated not as a race to patent, but as a collective pursuit for the betterment of all. The vision extends beyond individual discoveries to transform the entire ecosystem of biomedical research. By 2035, we envision a world where every researcher has access to the molecular tools needed to investigate any human protein, where AI-driven insights accelerate hypothesis generation, and where therapeutic breakthroughs emerge from truly global collaboration. The future of drug discovery is open, collaborative, and boundless. Trust Transparency Collaboration Shared Purpose Global Impact