Cell & Gene Therapy Insights

“Ultimately, the long‑term success of in vivo LNP delivery will depend on the ability to transform these platforms into robust, manufacturable, and globally accessible therapeutic modalities.” In recent years, the development of lipid nanoparticles (LNPs) including targeted LNPs (tLNPs) has positioned non‑viral delivery as one of the most important aspects of the evolving genetic medicines space for the delivery of nucleic acid‑based moieties.

“Rather than renting a therapeutic benefit on a month‑to‑month basis, we should be looking at it more as a long‑term investment – going from renting to owning.” Abi Pinchbeck, Commissioning Editor, Cell & Gene Therapy Insights , speaks with David Barrett, CEO, American Society of Gene & Cell Therapy, about the evolving role of the organization as the field it serves accelerates, the CGTxchange initiative to rehome deprioritized assets, and the urgent question of how reimbursement models must...

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Jude Samulski, Alumni Distinguished Professor, UNC Gene Therapy Center Jude Samulski’s Founders Award acceptance was a characteristically direct reflection on nearly five decades of AAV biology, and a candid assessment of where the field still has work to do. His central message: only 3% of AAV9 crosses the blood–brain barrier, and that figure demands a rethink.

Cell and Gene Therapy Insights 2026; 12(5), 503–507 10.18609/cgti.2026.058 Non‑viral delivery systems (such as lipid nanoparticles (LNPs), polymeric carriers, lipid‑polymer hybrids, etc.) are no longer only an enabling technology. They are becoming a big player in advanced and personalised medicines reaching patients at scale. As the complexity of the therapeutic systems increase, so does the inadequacy of the existing analytical and regulatory frameworks designed to describe and manage them.

Commentary Adrian Bot, Xianghong Li Bot and Li assess the rapidly expanding in vivo CAR ecosystem, reviewing lentiviral vector and LNP-RNA platform technologies alongside emerging clinical evidence and key safety considerations. Early results are encouraging, but the authors argue that in vivo approaches must still match the potency of optimized ex vivo products before meaningful displacement occurs.

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Decentralized manufacturing (DM) of advanced therapy medicinal products faces key challenges around quality consistency, supply chain coordination, and regulatory compliance across distributed sites. This article provides practical guidance for developers planning DM in the UK under the 2025 MM and POC regulatory framework.

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“We see in vivo as a much simpler and economical way to bring CAR‑Ts to patients, but it is as yet largely unproven.