Liver Diseases
At GENFIT, we take pride in our longstanding expertise in the field of liver diseases. We have established ourselves as a leader in understanding and developing therapeutic and diagnostic solutions for rare and life-threatening liver conditions with a specific focus on diseases with high unmet medical needs.
Before pivoting our focus to Acute-on-Chronic Liver Failure (ACLF) where the sense of emergency for patients, clinicians, regulators and payers is extremely high, our team of scientists previously unravelled the complexities of nuclear receptor signaling pathways and their involvement in liver diseases to successfully bring novel compound elafibranor (dual PPAR a/d agonist) from early-stage development to successful Phase 3 and precommercial readiness in Primary Biliary Cholangitis (PBC).
By leveraging our in-depth knowledge and experience, we strive to improve the lives of patients suffering from liver diseases worldwide.
Research
Our foundation is built upon a strong expertise in research. From understanding of molecular mechanisms of a disease and target identification and modulation, to establishing a network of experts, our talented and diverse team with various and complementary expertise and backgrounds continuously pushes the boundaries of scientific knowledge and employs state-of-the-art technologies such as human liver organoids and spheroids, to drive our research efforts forward.
Our research platforms can support the entire lifecycle of non-clinical and preclinical development, including in-vitro and in-vivo disease models. Our laboratory capabilities are comprehensive in the areas of pharmacology, molecular and cell biology, drug candidate screening, medicinal and analytical chemistry, biochemistry, OMICs analyses, and data science. Our in-house biobank allows us to offer unparalleled control and precision in handling and preserving biological samples, which are crucial for our research endeavors. We ensure the highest standards of sample quality, integrity, and accessibility.
We emphasize a data-driven and evidence-based approach, employing cutting-edge techniques to accelerate the drug discovery process and to develop breakthrough drug candidates. Our commitment to excellence fuels our quest for transformative therapies that address unmet medical needs, improve patient outcomes, and make a lasting impact on global healthcare.
Clinical Development
We have a breadth and depth of expertise in the design, conduct, and management of clinical trials, driven by many years of biotechnology/pharmaceutical industry experience. Our clinical trial protocols are developed through collaboration with KOLs, as well as input gathered during KOL Advisory Boards. We also take utmost care to include the patient voice in the design of our trials through close collaboration and partnership with patient advocacy groups.
Our success in clinical trial execution is driven by a robust global network of esteemed clinical research partners, including leading academic medical centers, hospitals, specialized clinics, and contract research organizations. These close collaborations allow us to ensure the highest quality of study conduct, all whilst adhering to rigorous regulatory standards and ethical guidelines.
Our extended team of clinical researchers and trial coordinators conduct global, multi-center trials with diverse patient populations, providing valuable insights into treatment efficacy, safety, and patient response across different demographics. As such, we are able to generate robust and comprehensive datasets that serve to strengthen regulatory submissions.
Data Science
Since its creation, GENFIT has seamlessly integrated data and IT technology into R&D scientific activities. Our data science expertise, combined with a deep understanding of drug discovery and development, accelerates research and optimizes clinical trials, reducing the time and cost of bringing new drugs to patients.
We leverage cutting-edge data management tools (graphs and lakehouse platforms) to efficiently organize and extract valuable insights from our vast and complex data repositories. Additionally, bioinformatic analysis methods are employed to explore and interpret internal and public OMICs data (microarray, RNAseq, etc.), identifying targets of small molecules and unraveling their mechanism of action.
In close collaboration with our scientists, we utilize machine learning techniques for in-depth data exploration and modeling (Machine Learning). This aids in understanding the factors influencing disease progression and identifying biomarkers for patient diagnosis or prognosis. We exploit artificial intelligence, (image processing tools and large language models) to create innovative tools that accelerate daily work and decision-making. Furthermore, we are actively advancing our digital transformation strategy for laboratory operations through the implementation and customization of an Electronic Lab Notebook and adhering the FAIR1 methodology.
Our teams are involved from the initiation of study design to biostatistical analyses to drive the R&D of different projects, from pre-clinical to clinical studies.
Regulatory Affairs
We possess a deep-rooted expertise in regulatory affairs, ensuring compliance with global regulations and facilitating timely submissions to health authorities. Our expertise comprises interactions with FDA/EMA for Investigational New Drug (IND) submissions, Breakthrough Therapy/Fast Track/Orphan Drug Designations, etc.
By collaborating closely with regulatory agencies worldwide, we navigate through the complex pathways of drug development and registration with attention to detail and a commitment to regulatory compliance. We strive to bring our therapies to commercialization, while maintaining the highest standards of safety and efficacy. Our dedicated team of regulatory affairs professionals closely monitors and interprets regulatory guidelines, staying up-to-date with the evolving landscape.
Market Access
Our objective is to bring our clinical candidates all the way up to commercial readiness, which means going beyond clinical and regulatory development to include market analysis and understanding of critical dynamics that shape commercial opportunities, from patient journey to payers’ approach and physicians’ intention to prescribe. Market studies and pre-marketing efforts are strategically designed to ensure that our therapies meet regulatory requirements, are effectively positioned in the market, and reach the intended patient population whether we go to market alone or with a commercial partner
We employ a comprehensive approach that encompasses quantitative and qualitative market research with key stakeholders, disease awareness and patient engagement, modeling and forecasting. By conducting in-depth market assessments, we gain valuable insights into patients’, clinicians’ and payers’ needs, treatment gaps, and emerging trends, enabling us to develop tailored strategies that resonate with healthcare providers and patients alike. Through planning and execution, we effectively communicate the value and benefits of our drug-candidates with the goal to optimize their potential to capture a significant part of the commercial opportunities we target.
1 FAIR: Findable, Accessible, Interoperable, Reusable