Therapy development is a central component of the Emma CPM catalyst wheel, focusing on creating new therapeutic options and translating research insights into actionable treatments for patients with rare diseases. This step involves identifying, optimizing, and validating therapeutic strategies through rigorous pre-clinical and clinical testing. By integrating cutting-edge technologies, fostering strategic collaborations, and adopting innovative approaches, we accelerate the development of therapies that address critical unmet medical needs.

We drive therapeutic innovation by utilizing patient-specific preclinical models (e.g., iPSCs, organoids), state-of-the-art technologies (e.g., gene editing, high-throughput screening), and forward-thinking valorization models. A key focus is the development of platform-based therapies, which move beyond the traditional one-disease-one-therapy paradigm. This approach enables scalable and versatile solutions to tackle multiple rare diseases more effectively. We also support a collaborative ecosystem to ensure that therapies are scientifically and regulatory robust, and tailored to patient needs, in alignment with the Emma CPM mission of advancing personalized medicine and improving outcomes for patients with rare diseases.

Key steps in the pre-clinical therapy development process that we focus on are: