Where Regulation Meets Innovation: Cytiva’s Playbook for Genomic Medicines
Genomic medicines now have a usable toolbox to silence, express, and edit genes, but cost, access, and CMC risk still slow progress. Cytiva’s team walked through concrete advances in AAV manufacturing, ex vivo cell processing, and lipid nanoparticle delivery, then showed how coordinated regulatory strategy can make personalized treatments practical.
Captured live at Advanced Therapies Europe, this workshop shows how engineered biology, modular automation, and platform regulatory strategies can shorten time to market while keeping quality defensible.
Session summary with Emmanuel Cameau, Scientific Director, Viral Vectors, Cytiva; and Anna Malinowska, Lead Regulatory Strategist, Biologics Manufacturing, Cytiva.
TL;DR In a hurry? Here are the essentials at a glance:
Stabilize the biology first. Engineered AAV producer cell lines can lift titers, improve packaging, and reduce encapsidated host-cell DNA to tighten CQAs.
Model cost with real scales. In a 2,000 L scenario targeting ~2 × 10^19 vg annually, stable AAV lines showed significant COGS advantages even with a lower full:empty ratio.
Standardize ex vivo. A modular, closed, digital platform for isolation-to-fill cuts labor, floor space, and batch failure risk while preserving product phenotype across variable inputs.
Use LNPs where they shine. Ex vivo LNP kits enabled efficient T-cell dual editing with maintained viability, preserved killing, and ~50% less hands-on time versus electroporation.
N-of-1 is real when platforms exist. The KJ case was enabled by a pre-built CRISPR “cookbook” and cross-institution collaboration, compressing diagnosis-to-dose to six months.
Regulators are enabling. FDA guidance, expedited pathways, platform submissions, and clear long-term follow-up expectations can accelerate safe access if engaged early.
Engineer the biology, then scale
AAV production routes—transient transfection, packaging/producer lines, baculovirus—each leave gaps. The Cytiva thesis: start by engineering the line so biology is predictable, then scale. That means raising titers, tuning packaging, and integrating full expression cassettes at defined loci so lines behave like true producers. A near-term win is quality—targeting host pathways that fragment DNA to reduce encapsidated host-cell DNA that downstream cannot remove.
“We have a toolbox to silence, express, and edit genes.” — Emmanuel Cameau, Cytiva
Make that toolbox pay off by locking CQAs upstream: choose higher-producing hosts, optimize helper/rep/promoter architecture, and edit the line (e.g., DNA-fragmentation factors) to lower hard-to-purge impurities.
Stabilize AAV supply and cost with producer lines
Stable producer lines change the cost curve at commercial scale. In Cytiva’s model at 2,000 L to supply roughly 2 × 10^19 vg annually, a stable process needed far fewer runs and delivered a marked COGS reduction versus transient—even with a lower full:empty ratio today. As fullness improves, the gap widens.
“The cost difference at scale is really high in favor of a stable process, even before fullness is fully optimized.” — Emmanuel Cameau, Cytiva
Plan accordingly: invest in the line, validate with realistic demand, and let downstream work with a cleaner input. Where licencing and IP apply, model those explicitly; the direction of travel still favors stability.
Modernize ex vivo manufacturing
Autologous workflows remain complex and labor-heavy, with non-trivial batch failures. Cytiva’s modular platform links a short-use isolation/harvest/formulation unit with an expansion unit for activation, gene transfer, and growth, orchestrated by one digital layer. The goal: reproducible phenotypes after magnet-based selection across variable starting material, lower labor, fewer assets, smaller footprint, and more doses per suite.
“A fully automated, modular platform can run from isolation to formulation and cut labor and footprint while holding phenotype.” — Emmanuel Cameau, Cytiva
Make orchestration the default: close the process, digitize records, and design for quick tech transfer so the same playbook runs from PD bench to GMP floor.
Go non-viral where it helps
With Precision NanoSystems as Cytiva’s LNP center of excellence, ex vivo kits enabled CAR expression during expansion and efficient dual knock-outs in T cells with preserved viability, no off-targets detected in the data shared, and about half the hands-on time versus electroporation—while maintaining cytotoxic function.
“Dual-edited T cells were produced more efficiently with LNPs than with electroporation, with viability and function preserved.” — Emmanuel Cameau, Cytiva
Pick the right jobs for LNPs: edits that benefit from gentle delivery, programs constrained by EP throughput, and workflows where shaving 50% handling time creates real capacity.
Make N-of-1 therapies programmatic
The KJ pediatric case shows a platform beats heroics. A pre-built CRISPR “cookbook” from the IGI–Danaher Beacon collaboration plus aligned clinical and development sites enabled in vivo liver editing via LNP in roughly six months from diagnosis.
“The entire process from diagnosis to treatment took just six months because the platform and partners were already in place.” — Anna Malinowska, Cytiva
Treat N-of-1 like a product line: reusable designs, common analytics, preset handoffs, and partners ready to execute. That’s how one case becomes a pipeline.
Design trials for rare reality
When n is tiny or equals one, adaptive designs, antisense modalities, LNP-enabled editing, liquid biopsies, and platformized CMC make evidence generation feasible. The harder parts sit outside the lab: early agency dialogue, reimbursement models, and ethics for pediatric and critically ill patients—without sacrificing rigor.
“N-of-1 means the patient becomes their own control, but statistical rigor and clear methodology still apply.” — Anna Malinowska, Cytiva
Write the playbook up front: define endpoints that regulators accept, pre-agree sampling and analytics, and establish long-term follow-up plans that match risk.
Work with regulators as enablers
FDA is signaling how to move faster safely: expedited pathways with appropriate surrogates in serious conditions, clearer guidance spanning discovery to manufacturing, pilot support for rare disease programs, and openness to platform submissions and cross-program data reuse. Long-term follow-up expectations remain stringent for gene therapies, with willingness to revisit as evidence grows.
“Regulatory frameworks, with the right approach, can foster scientific progress rather than slow it down.” — Anna Malinowska, Cytiva
Engage early, document mechanism, and use platform data to justify what you test—and what you don’t.
Collaborate end-to-end
Cytiva’s model ties R&D, analytics, GMP manufacturing, digital, and regulatory into a single line of sight, from academic concept to commercial supply. Recent collaborations span the Beacon platform, the KJ program, and a rapid build-out of a state-of-the-art CAR-T facility—using automation, facility design, and standardization to pull time out of tech transfer.
“Alone we can do so little. Together we can do so much.” — quoted by Anna Malinowska in closing
Make collaboration the operating system: shared playbooks, shared data, shared speed.
Final word
Make biology stable, make processes modular, and make regulation a design input, not an afterthought. Do this and genomic medicines move from fragile pilots to reliable access—even when the patient is one child.
Keep the momentum: join the community at Advanced Therapies Week 2026 in San Diego, February 9 to 12, 2026.
)