When Michelle Werner received the devastating news that her son Caffrey had been diagnosed with Duchenne muscular dystrophy (DMD) five years ago, she was plunged into a world of uncertainty and isolation. As the CEO of Alltrna and a former Big Pharma executive with extensive experience in drug development, Werner found herself at a crossroads. The journey with her son’s rare disease not only brought immense challenges but also ignited a passion for driving change in the field of rare disease research and development.
DMD is a progressive disease that often leads to patients becoming wheelchair-bound and facing grim prognoses. Despite advancements in treatment, the standard of care for DMD has remained largely unchanged for decades. Caffrey’s diagnosis at the age of 10 did not align with the typical age range for clinical trial eligibility, highlighting the limitations and inadequacies in the rare disease R&D pipeline.
Werner’s deep understanding of the drug development process and her personal experience with her son’s diagnosis prompted her to seek new approaches to addressing the challenges in rare disease research. She realized the urgent need for innovative treatments that could target multiple rare genetic diseases simultaneously, rather than addressing them one by one.
In 2022, Werner made a significant career shift from the realm of Big Pharma to the innovative landscape of biotech. Joining Alltrna as CEO, she spearheaded the development of tRNA technology aimed at reversing mutations associated with codon diseases. This technology offered a promising solution for treating a wide range of rare diseases with a single tool, presenting a groundbreaking approach to addressing the complexities of rare genetic conditions.
One of the key strategies Werner introduced at Alltrna was the concept of basket trials, commonly used in oncology, to treat patients with the same mutation in a disease-agnostic manner. This approach allows for a more inclusive and efficient way of conducting clinical trials, encompassing both common and ultra-rare populations.
Balancing her roles as a mother and a CEO, Werner found that her dual perspectives enriched her approach to addressing the unmet needs in rare disease R&D. Drawing from her experience in Big Pharma, she emphasized the importance of taking risks in pursuit of scientific advancements and prioritizing the well-being of patients over corporate interests.
As Caffrey continues his journey with DMD, Werner remains committed to driving progress in the field of rare disease research. With new treatments on the horizon and a growing focus on innovative therapies, there is hope for improving the lives of patients like Caffrey. Werner’s dedication to making a difference in the lives of those affected by rare diseases serves as a beacon of hope for the future of healthcare.
In conclusion, Michelle Werner’s transformative journey from a Big Pharma executive to the CEO of Alltrna reflects her unwavering commitment to revolutionizing rare disease research and development. Through her innovative approach and dedication to improving patient outcomes, Werner stands at the forefront of a new era in biotech innovation.
