BIO
Gene therapy's technological journey from upstream to downstream
The number of approved gene therapies for the treatment of monogenetic disorders and diseases is increasing and significant advances have been made in the development of revolutionary new vector, manufacturing and analytical technologies.
The strategic adoption of technological trends across upstream and downstream processes (USP and DSP) stands as a defining factor in improving safety and efficacy while reducing production costs. Biopharma companies must embrace these trends and effectively integrate USP and DSP technologies into their current systems to unlock the full potential of gene therapies.
Advances in upstream innovation
Design and optimisation of USP stand as the cornerstones for forging successful gene therapies, and groundbreaking innovations are shaping the future. Genetic engineering breakthroughs give rise to synergy with biotechnology that elevates yields, diminishes production expenses, and amplifies scalability.
Developing downstream dynamism
As with USPs, there are also many technologies set to elevate DSP in gene therapy development and manufacturing. These advances in DSP technology aim to reduce levels of process-related contaminants for the safety, quality and stability of the final product while also increasing recoveries and achievable titres.
The challenges of manufacturing gene therapies
Although the progress in gene therapies is an exciting era in healthcare, it is essential to acknowledge and tackle the challenges of planning and carrying out gene therapy manufacturing. These challenges predominantly surround scalability, stability, regulatory compliance, and access to patients.
Gene therapy stakeholders must stay attuned to advancing technologies in USP and DSP, adapting as needed. Collaborating closely with manufacturing partners, efforts to reduce costs remain imperative to ensure commercial feasibility and make transformative treatments accessible. Furthermore, the dynamic landscape of gene therapies will drive ongoing refinement of regulatory guidelines, mirroring the rapid scientific progress and evolving market dynamics, ultimately advancing the journey toward revolutionary healthcare solutions.
Pharmaceutical Review (EPR) magazine, you can read it in full on pages 44-46 by clicking the button below.