The patient connection – How CDMOs enable patient-centric solutions

March 5, 2025 

In today’s rapidly evolving biotech landscape, patient-centricity has become more than just a buzzword—it’s a driving force behind innovation in drug development and manufacturing. For CDMOs, this means going beyond traditional production roles to become enablers of therapies tailored to individual patients or specific populations.

Personalized RNA-Based Therapies

Personalized medicine, particularly in the RNA space, is reshaping how we approach treatment:

• Custom-Tailored Therapies: RNA-based platforms, like mRNA vaccines or therapies, can be designed to target specific mutations or disease pathways unique to individual patients.
• Flexible Manufacturing Capabilities: CDMOs play a pivotal role by developing flexible, small-scale production lines capable of handling highly customized RNA therapies.
• Case Example: Imagine a patient with a rare genetic disorder—RNA-based therapies can be designed to silence or correct the defective gene, and CDMOs ensure the rapid production of these highly specific treatments under GMP conditions.

Compassionate Use Programs: Scaling for Urgency

Compassionate use, also known as expanded access, is a lifeline for patients who have no other treatment options. Here’s how CDMOs are enabling rapid response in these critical scenarios:

• Accelerated Manufacturing Timelines: CDMOs with agile processes and cutting-edge facilities can quickly scale production to meet urgent needs without compromising quality.
• Balancing Speed and Compliance: Producing therapies for compassionate use requires navigating stringent regulatory requirements while delivering within tight timelines—a challenge CDMOs are uniquely equipped to handle.
• Real-World Impact: For example, during the pandemic, the rapid production of RNA vaccines was made possible by CDMOs scaling manufacturing at unprecedented speeds to meet global demand. This same expertise can be applied to individual patients in critical need.

Key Enablers of Patient-Centric Solutions

To support personalized therapies and compassionate use programs, CDMOs are focusing on:

1. Innovation in Processes: Developing modular and multi-product facilities that can switch between products efficiently to meet personalized demands.
2. Digital Integration: Using AI and automation to improve precision, track production in real-time, and ensure scalability for niche treatments.
3. Collaborative Partnerships: Working closely with biotech innovators, regulators, and healthcare providers to align manufacturing strategies with patient needs.

The Impact on Patients

By enabling patient-centric solutions, CDMOs are helping to:

• Bring Hope to Rare Disease Patients: Accelerate the development of targeted therapies for diseases with small patient populations.
• Improve Accessibility: Reduce production times so that therapies can reach patients faster, even in regions with limited healthcare infrastructure.
• Support Long-Term Innovation: Lay the groundwork for the future of personalized medicine, ensuring that treatments are not just effective but also accessible when and where they are needed

At Exothera, we are committed to a patient-first approach by delivering scalable, flexible, and high-quality manufacturing solutions. With cutting-edge technologies like the Ntensify platform, we empower biotech innovators to accelerate personalized therapies and compassionate use programs efficiently and compliantly.

Authors: Barbara Cornelissen, Marketing Director, Exothera