Cell & Gene Therapy Insights

Lipid nanoparticles (LNPs) have emerged as the predominant delivery platform for nucleic acid therapeutics, enabling transformative clinical applications ranging from prophylactic mRNA vaccines to in vivo gene editing, gene therapy, protein replacement therapies, and personalized cancer immunotherapies [1–4]. As LNP formulations become increasingly complex, incorporating novel ionizable lipids, targeting ligands, and multi-component payloads—the analytical challenges expand proportionally.

Novartis paused eight cell therapy trials in autoimmune and neurological disease after three deaths [1] Novartis paused eight clinical trials of rap-cel, an experimental CAR-T cell therapy targeting autoimmune and neurological disorders, after three patients died. The halt, implemented on August 24, 2026, followed three cases of immune effector cell-associated hemophagocytic syndrome, a severe, life-threatening immune reaction.

Confirming product identity, genome integrity and freedom from adventitious agents requires a single test to hold up across genomes as small as an AAV vector and as large as a human genome, against regulatory guidance that continues to evolve (ICH Q5A(R2)). Here is how FUJIFILM Biotechnologies built validated NGS workflows covering library preparation, genome integrity, sensitivity testing and adventitious agent detection, cutting biosafety testing turnaround from up to 8 weeks to 1–2 weeks.

Triple transfection constrains AAV manufacturing on multiple fronts: yield, scalability, quality, and cost per dose. In this webinar, UMass Chan Medical School and OXGENE®, a Minaris brand, present data on TESSA® a plasmid-free production platform that has delivered up to 40x higher AAV yields and up to 80% full capsids pre-enrichment, compared with roughly 40% for triple transfection.

01 How combining fluorescence-based single cell dispensing (Pala) with automated capillary western immunoassay (Leo) addresses two major bottlenecks in iPSC CRISPR engineering: single cell cloning and functional knockout confirmation 02 Why IFNγ-induced surface β2M expression improves fluorescence-based enrichment of edited clones, increasing knockout recovery to 62% versus 40% in untreated cultures 03 How Simple Western analysis discriminates partial from complete β2M knockout and detects...

“Our journey has taught us that the most important advances in gene editing are not measured by editing efficiency or novel molecular tools, but by our ability to translate those innovations into therapies that address unmet patient needs." Over the past decade, gene editing has transitioned from scientific promise to therapeutic reality, creating new opportunities to address previously untreatable diseases.

“...collaborative, public–private approach ensures that the next generation of genomic therapies can successfully transition from bespoke treatments to accessible, scalable medicines.” Genome editing drugs are rapidly moving into the clinic, and the prospect of individualized CRISPR drugs has demonstrated clinical promise, transitioning rapidly from design to infusion into patients. New initiatives build on the precedent set by individualized antisense oligonucleotide therapies.

Cell and Gene Therapy Insights 2026; 12(5), 663–680 10.18609/cgti.2026.080 Gene therapy has traversed a long journey, starting as a theoretical concept, progressing to a controversial therapeutic option, and further on to becoming a promising cure for multiple diseases. One of the major bottlenecks in this journey has been the availability of suitable delivery vectors. Since viruses possess the natural ability of cellular invasion, they have been the first choice for such endeavors.

“We argue the case against the use of HHGE for at least the next 10 years for three central reasons: lack of medical need, unproven safety, and significant unresolved ethical and governance concerns” Multiple entities have argued against the use of heritable human germline editing (HHGE) for at least the next ten years due to a lack of medical necessity, unproven safety, and unresolved ethical and societal concerns.

“Five years from now, when we have treated and cured many people with sickle cell disease, it will begin to feel mundane.” Abi Pinchbeck, Editor, Cell and Gene Therapy Insights , speaks with Paula Cannon, Distinguished Professor of Molecular Microbiology and Immunology, Keck School of Medicine of USC, about the evolution of gene therapy from a bold engineering challenge to an increasingly routine clinical reality.