Construction of pipelines for metabolomics analyses, in silico genetic conservation, and new techniques for evaluating the safety and efficacy of drugs in Zebrafish embryos, aiming at the expansion and scalability of the global technological platform

• Grant number: 24/10756-0
• Support Opportunities: Research Grants - Innovative Research in Small Business - PIPE
• Start date: July 01, 2025
• End date: June 30, 2027
• Field of knowledge: Biological Sciences - Pharmacology - Cardiorenal Pharmacology
• Principal Investigator: Anna Laura Viacava Américo
• Grantee: Anna Laura Viacava Américo
• Company: Fiorino e Americo Consultoria Ltda
• CNAE: Atividades profissionais, científicas e técnicas não especificadas anteriormente
• City: São Paulo
• Pesquisadores principais: Patrícia Fiorino
• Associated researchers: Bianca Helena Ventura Fernandes ; Cynthia Rodrigues Muller
• Associated scholarship(s): 25/11106-1 - Construction of pipelines for metabolomics analyses, in silico genetic conservation, and new techniques for evaluating the safety and efficacy of drugs in Zebrafish embryos, aiming at the expansion and scalability of the global technological platform, BP.PIPE

Abstract

In Brazil, there has been a significant increase in companies dedicated to research, development, and innovation (R&D&I) to discover new molecules with therapeutic properties. According to "Valor Inovação Brasil 2023," the five largest pharmaceutical companies (Aché, Eurofarma, Roche, Hypera Pharma, and ABBVIE) invested more than R$1.8 billion in R&D&I for new "hits" or "leads" molecules for the treatment of economically relevant diseases. In this context, both incremental and radical innovations are important for the development of new drugs. Developing a new drug takes 10 to 15 years and costs around $2.5 billion, with only 4.9% of molecules in development reaching the market.In interviews with pharmaceutical industry experts, we confirmed that the biggest challenges in the R&D&I process are the time and high cost of bringing a new drug to market. We identified a growing demand for processes that optimize and reduce R&D&I time, increase the speed of patent registration, competitiveness, and success rate, and lower costs.Focusing on this demand, our startup proposes modifying the current pipelines, offering the market an innovative approach, focusing on safety and efficacy evaluation in the pre-clinical phase through an optimized high throughput screening platform using Zebrafish (ZF) embryos, enhancing existing methodologies to achieve faster and more accurate results. In this context, based on the results of Phase 1 of PIPE, we developed and validated cardiovascular and cardiotoxicity functional analyses to increase the accuracy in defining promising molecules by excluding those with potential adverse effects or low efficacy and prioritizing those with higher chances of success in R&D&I.Our main differentiator is the efficient implementation and applicability of the platform to accelerate the R&D&I of new drugs, as well as the translation of cutting-edge methodologies to market services, allowing scientific advancements to quickly reach society. There are two competitors in the European market with similar solutions, which reinforces the potential of our proposal and market interest, as it validates the feasibility of our business model, indicating that we are aligned with global research and development trends. This scenario confirms the relevance and opportunity to expand our technological platform, highlighting its applicability and impact on scientific and technological advancement.Another evidence of the feasibility of our proposal was demonstrated through interviews with pharmaceutical industry experts, who revealed a strong interest in implementing our technological platform to optimize specific processes, which validated our entry market (beachhead market) as being the pharmaceutical industry and biotechnology companies that synthesize new molecules. However, besides the pharmaceutical sector, we identified interest from four other sectors: Food and Nutrition, Cosmetics, Agrochemicals, and Basic Research, highlighting the potential scope and use of our platform. Despite the initial focus on the pharmaceutical market, diversifying customer segments favors growth in different areas, contributing to the company's long-term financial stability.Another aspect that reinforces the innovative potential of our platform was the Patent Feasibility Study, conducted by Baltazar Intellectual Property and Technology Inova Simples, which confirmed the possibility of patenting our process, demonstrating the originality and robustness of our proposal. Thus, the PIPE Phase 2 project is essential to expand and consolidate our model, which offers a broad, customizable, robust, and innovative portfolio with potential applicability to various market segments, initially focusing on the pharmaceutical and biotechnology markets. (AU)