Some individuals drift into science; others are driven by profound purpose. Dr. Dima Martini-Drew, founder of CortexBio Ltd., embodies both curiosity and experience. Her journey began in Aleppo, where a formative encounter with a young girl suffering from an unnamed disease planted the seeds of her lifelong mission. Years later, while working with Genzyme, she identified the girl’s condition as MPS I—a revelation that shaped her scientific calling.
Encounters with patients like the youngest ever diagnosed with Pompe disease deepened her resolve. These experiences became the foundation for her vision: to unravel the “metabolic mosaic” connecting rare childhood diseases and the complexities of aging, and to translate science into timely, life-saving action.
Reconceptualizing Metabolism: A Groundbreaking Framework
CortexBio was founded on the revolutionary premise that rare metabolic diseases and aging are interconnected threads of the same metabolic story. Dr. Martini-Drew’s “metabolic mosaic” hypothesis challenges conventional boundaries, presenting aging as an inherited metabolic disorder that shares mechanisms with rare inborn errors. This perspective reframes aging not as a slow decline but as a dynamic metabolic imbalance—one that can be comprehended, anticipated, and potentially mitigated.
This systems-based approach is reflected in CortexBio’s proprietary platforms, multi-omic research models, and strategic collaborations, all designed to transform metabolic insight into early, precise, and scalable interventions. At its core, the goal is to architect new possibilities for those who have long remained underserved by traditional frameworks.
Bridging Science and Human Experience
Dr. Martini-Drew’s work synthesizes decades of research, mapping connections between energy metabolism, cellular respiration, and nutrient sensing to provide actionable insights for drug repurposing and therapeutic development. Her strategy draws on multi-omics data, knowledge graphs, and adaptive clinical trials, always grounded in the lived experiences of patients and their families.
She is a strong advocate for including caregiver outcomes in clinical trial design, helping shift the definition of therapeutic success toward more compassionate, comprehensive care. By integrating caregiver well-being into regulatory submissions and trial protocols, Dr. Martini-Drew expands the conversation around healthcare value, ushering in a new model of patient-family-centered innovation.
Leadership: Vision, Empathy, and Collaboration
As founder and CEO, Dr. Martini-Drew leads CortexBio with vision, empathy, and a collaborative spirit. She unites scientists, clinicians, patients, and regulators, fostering an environment where innovation thrives. Her leadership is defined by a focus on blind spots in science, overlooked patient needs, and the gaps in how the world responds to rare diseases.
Moreover, Dr. Martini-Drew is recognized for dismantling structural barriers, such as fragmented research and regulatory delays, ensuring that no patient is left behind. “Innovation begins where systems fail, and we have the responsibility to design what comes next,” she says.
Building Excellence through Collaborative Teams
While Dr. Martini-Drew’s vision drives CortexBio, the company’s success is determined to reflect the collective expertise of a multidisciplinary team spanning computational biology, clinical research, regulatory affairs, and patient advocacy. The organization fosters a culture where diverse scientific perspectives converge to tackle complex metabolic challenges, ensuring that innovation emerges from collaborative excellence rather than individual effort alone
Innovation for the Future of Healthcare
Dr. Martini-Drew transforms complex challenges into catalysts for innovation. The Metabolic Synergy Tool, CortexBio’s flagship platform, integrates multi-omics data to map metabolic connections between rare diseases and aging. It identifies shared therapeutic vulnerabilities that are invisible to siloed research, enabling single interventions to span multiple diseases, age groups, and markets.
By leveraging knowledge graphs, patient registries, and Genome-Wide Association Studies (GWAS) datasets from conditions like Parkinson’s and dementia, the platform identifies shared therapeutic vulnerabilities previously hidden from view. Through adaptive trial designs, including platform trials and multi-arm models, CortexBio accelerates therapeutic development, reduces timelines by 25%, and cuts development costs by 15%.
This model allows single interventions to serve multiple patient populations, reshaping both the economics and accessibility of rare disease research.
Measurable Progress in Metabolic Medicine
CortexBio’s impact extends beyond individual success stories to measurable outcomes across the rare disease landscape. Since its founding in September 2024, the company has established partnerships with leading academic institutions and regulatory bodies, with early-stage collaborations focusing on platform trial designs that could reduce development timelines by 30-40% compared to traditional approaches.
The Metabolic Synergy Tool has analyzed over 1,000 metabolic pathway connections, identifying 15 potential therapeutic targets for cross-indication development. These quantitative markers demonstrate the tangible progress toward Dr. Martini-Drew’s vision of transforming rare disease therapeutics.
Fostering a Collaborative Ecosystem through Strategic Partnerships
At the core of CortexBio’s mission is collaboration. The organization works extensively with academic researchers, clinicians, regulators, and patient advocates, uniting diverse stakeholders around common objectives. This commitment to partnership is exemplified through strategic alliances with registries and various patient advocacy groups dedicated to metabolic diseases. These collaborations seek to produce meaningful outcomes, including the development of caregiver-reported outcome measures that assess caregiver burden alongside traditional clinical endpoints.
Such efforts aim to inform regulatory submissions and enhance clinical trial design. A notable example is the partnership with the Critical Path Institute, demonstrating how industry, academic, and regulatory entities can combine efforts to accelerate therapeutic development while upholding scientific integrity. Engagement with patient organizations for Inherited Metabolic Diseases continually grounds Dr. Martini-Drew in real-world experience, ensuring the pursuits remain patient-centered and impactful.
Transforming Lives through Early Intervention
Dr. Martini-Drew’s proudest achievements are found in renewed hope for patients, such as the youngest recipient of enzyme replacement therapy for Pompe disease. Her pioneering work demonstrates that timely intervention can save lives and catalyze global changes in care standards.
Research now confirms that, by initiating treatment just 18 hours after birth, the child achieved normalized heart function in 21 weeks and typical neurodevelopment by 46 weeks. This case catalyzed global changes in newborn screening protocols, affirming that time-sensitive care can save lives.
The Road Ahead: Building the Future, One Mosaic at a Time
Looking forward, Dr. Martini-Drew is developing the Metabolic Synergy Tool and advocating for global platform trials that cross traditional boundaries. She is also writing Unraveling the Metabolic Mosaic, a manifesto and roadmap for the future of metabolic medicine.
She envisions a future where therapeutic development transcends silos, enabling continuous learning across diseases, ages, and populations—a living model for global precision medicine.
Her mission: to bring the full picture of metabolic health into view and ensure that scientific innovation swiftly translates into patient benefit.
Navigating Complex Regulatory and Scientific Landscapes
CortexBio, as an innovative start-up, is dedicated to transforming the landscape of metabolic disease treatment. The company’s approach focuses on utilizing accelerated approval pathways and adaptive trial designs to break down existing barriers in drug development. By collaborating closely with regulatory agencies, CortexBio strives to set new standards and drive scientific progress, ultimately enhancing patient outcomes and offering hope to those affected by both rare and common conditions. The company aims to leverage learnings from rare disease and aging research to benefit seemingly unrelated conditions simultaneously, using regulatory pathway innovation to genuinely serve underserved patient populations.
Balance, Curiosity, and Connection
Dr. Martini-Drew creates balance through curiosity and connection, finding inspiration in both her scientific pursuits and life’s quiet moments. Time with family, especially newborns, reminds her that the metabolic mosaic is not abstract—it is alive in every patient. “Each child is a clean slate, a brilliant genius with an intricate metabolic map,” she reflects.
Ensuring Inclusive Innovation in Rare Disease Research
CortexBio is committed to addressing the historical lack of diversity in rare disease clinical trials, recognizing that genetic diversity is crucial for understanding metabolic disorders across different populations. The company actively partners with patient advocacy organizations across Europe and beyond to ensure that clinical trial designs accommodate diverse cultural contexts, language barriers, and socioeconomic factors that can limit participation. Dr. Martini-Drew emphasizes that true innovation in metabolic medicine requires representation from all affected communities, particularly underserved populations who have historically been excluded from rare disease research.
This commitment extends to fostering diversity within the scientific team and ensuring that research findings benefit patients regardless of background or geographic location. Having worked in the Middle East, Dr. Martini-Drew has firsthand experience with discrimination between patients from rich and poor countries, which further informs the efforts to promote inclusivity and equitable access in clinical research.
A Message Woven from Science, Scars, and Hope
Dr. Martini-Drew believes that biology carries the echoes of memory and that empathy is a transformative force. Her leadership is guided by the conviction that the most profound solutions emerge when we bridge what appears unconnected.
She reflects: “No scientific breakthrough, no matter how extraordinary, exists in isolation. It must serve life. It must elevate dignity.” She encourages others to remain curious, collaborate passionately, and embrace the possibilities ahead.
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