2025 cell and gene challenges: Thoughts from our editorial board

Creating a seamless, scalable infrastructure
In 2025, the biggest challenge facing the CGT sector will be improving patient access through more efficient and scalable manufacturing solutions. Traditional centralized manufacturing models have proven inefficient, with long delays between approval and patient treatment. To address this, decentralized and point-of-care manufacturing approaches offer solutions by bringing production closer to the patient. However, these models still face significant hurdles, such as ensuring consistency in quality control and maintaining regulatory compliance. While the FDA and MHRA are making progress in defining these new manufacturing models, the industry must overcome challenges in standardizing processes across different sites. As we strive to get therapies to patients faster, the key will be finding ways to overcome these bottlenecks and create a seamless, scalable infrastructure for delivering advanced therapies at the point of care.
• Carol Houts, Chief Strategy and Commercial Officer, GermFree

Integrating new tech into manufacturing and QC testing
The huge promise of CGT to treat serious, and often life-threatening diseases has so far not achieved widespread commercial success due to challenges in reliable GMP manufacturing and high costs of QC testing. Industry’s efforts to improve manufacturing processes by including new technologies and automation will determine the viability of CGT as next generation medicine. Adopting technological innovations, however, brings challenges in demonstrating and achieving product quality. Quality risk assessment, along with QbD tools, will help establish the control strategy for new processes. Reliable quality systems with QC strategies and method suitability will ensure quality and regulatory compliance are achieved. Integration of new manufacturing technologies under such quality management measures can ensure product success, manufacturing efficiency improvement, and cost reduction.
• Claudia Lin, Ph.D., Executive VP, Pharmatech Associates – a USP company

Manufacturing costs and high price tags
The biggest challenge facing CGT is the high price of manufacturing that makes products so expensive that it limits patient access. Biotech companies also struggle to raise enough money to pay for preclinical and early clinical development, due to the high per-batch cost in GMP. It is critical for industry, academia and regulators to partner together and find new, less costly ways to manufacture and release CGT products. These methods must result in products that maintain the same (or better) safety and efficacy.
• Lara Ionescu Silverman, Ph.D., Principal Consultant, LIS BioConsulting

Addressing scalability components
Scalability will become an even greater challenge to the industry going forward: As CGT products continue to penetrate into earlier line treatments and larger patient populations, addressing the key components of scalability (cost, capacity, and skilled workforce) will be essential to fuel further sector growth.
• Sanjin Zvonić, Ph.D., Sr. VP, Business Development & Practice Expert, Dark Horse Consulting

Enabling widespread access
Patient access continues to be a huge challenge. High treatment costs, resulting from expensive and complex manufacturing processes, create supply chain bottlenecks and delays in turnaround time. Regulatory hurdles can further limit availability. Clinician awareness and willingness to prescribe, specialized infrastructure, and trained personnel are required for administration, which can be scarce in underserved areas. Additionally, not all patients meet the strict eligibility criteria, further narrowing the patient pool. Lastly, reimbursement policies are still evolving, and insurance coverage is inconsistent, making access difficult for many. These factors combined hinder widespread access to CGTs.
• Mark Flower, VP of Business Development, Cryoport Systems

Read challenges related to:
•Investment and market; technology and innovation
•Scalability and manufacturing; global accessibility; supply chains