Non‑viral gene editing is emerging as a compelling alternative to viral vector–based gene therapy for hematopoietic stem and progenitor cells (HSPCs), offering the potential for precise, targeted gene insertion while avoiding the manufacturing complexity, payload limitations, and cellular stress often associated with viral donors. However, achieving efficient homology‑directed repair (HDR) in HSPCs while preserving cell viability, fitness, and long‑term function remains a key challenge.

In this webinar, we explore recent advances in non‑viral knock‑in strategies using Touchlight’s mbDNA™ (MegaBulb DNA)—an enzymatically produced, circular single‑stranded DNA template designed to enable highly efficient HDR with minimal toxicity. Presenters will share data demonstrating robust targeted integration in primary human CD34⁺ HSPCs across a range of mbDNA doses, using co-electroporation with CRISPR/Cas9 RNPs and compared to AAV and plasmid DNA controls. These results highlight unrivaled knock‑in efficiencies alongside preserved cell viability and recovery, underscoring the potential of mbDNA as a next‑generation HDR donor format to replace the incumbent viral vectors. Studies evaluating the impact of this system on the engraftment potential of edited HSPCs are currently ongoing. The speakers also outline how mbDNA can be combined with emerging lipid nanoparticle (LNP)-based delivery systems, pushing the frontiers of HSPC gene editing.

The session also discusses how non‑viral mbDNA‑based platforms can be integrated with emerging genome engineering and delivery strategies to enable safer, scalable, and lineage‑oriented gene therapy workflows. Register to watch the session and gain practical insight into how non‑viral HDR donor formats can help overcome key limitations of viral approaches and support the development of more precise, effective therapies for inherited hematopoietic diseases.

Key learning objectives:

• Learn why non‑viral gene editing is gaining momentum for use in blood stem cells and how it compares to traditional viral approaches
• Discover how mbDNA™ can be used to insert genes more precisely into human stem cells while maintaining cell health and viability
• Explore how new non‑viral gene editing and delivery strategies could enable safer, more scalable gene therapies for inherited blood disorders

Watch the webinar to see the data in action.