Developing Exosome-Based RNA Therapeutics For CNS Regeneration

A conversation with Lior Shaltiel, Ph.D., CEO and Director, NurExone Biologic

As exosomes have emerged as a next-generation therapeutic platform for multiple diseases, leaders in the RNA community are now focusing on these nanoparticles as the ultimate drug delivery system for a wide range of indications from oncology to dermatology. NurExone has taken a unique approach and is using exosome therapies for regenerative medicine to develop promising therapies for central nervous system (CNS) injury. In this Q&A, Advancing RNA’s Acquisition Editor Michael Soloway caught up with NurExone Biologic’s CEO, Lior Shaltiel, Ph.D., to discuss the company’s approach to RNA therapeutics.

NurExone is not the first company in the field of exosomes. What does the current exosome landscape look like? What are the most recent scientific advancements in the field and how do they affect your approach?

The exosome field is growing quickly, with companies focusing on applications from oncology to aesthetics to longevity. NurExone focuses on the central nervous system, where we are developing a minimally invasive therapy that achieves a broad regenerative effect rather than a local or surface-level one.

Recent scientific work highlights the importance of understanding the source of the exosomes, since the properties of the cells they come from directly affect therapeutic potential. This knowledge is critical for both efficacy and regulation, which is why we work with well-characterized and GMP human bone-marrow-derived mesenchymal exosomes.

At the same time, regulatory developments in the United States are beginning to clarify the path for exosome-based aesthetics. This helps separate serious, compliant pharmaceutical innovation from the unregulated exosome wellness products that currently dominate the market.

Why are these advancements so important to the exosome field as a whole and what role do they play in how CNS injuries are treated?

Greater scientific focus on exosomes is drawing more attention from regulators and accelerating the creation of clearer FDA guidance. This progress is helping the entire field move from experimental use toward recognized, regulated therapeutics. As exosome science becomes better understood, it strengthens confidence in the technology and opens the way for clinical development in complex conditions such as CNS injuries, where repair was once thought impossible.

For example, we are developing a biological exosome-based technology drug platform, using extracellular vesicles (EVs) specifically designed for CNS applications. Our proprietary ExoTherapy platform leverages the natural properties of exosomes to target damaged neural tissue. Our exosomes can be loaded with a wide range of molecules, such as siRNA, to enhance their inherent therapeutic potential.

We are also developing our lead product, ExoPTEN, as a novel therapy for acute spinal cord injuries and other central nervous system indications. After a brief minimally invasive ExoPTEN treatment cycle, we have observed a 75%-100% recovery of motor function, sensation, and bladder control in the animals we studied.

Exosomes are still a developing delivery modality. What are the unique challenges of sourcing and/or manufacturing exosomes?

The main challenge is repeatability. Unlike small molecules, exosomes are complex biological entities that cannot be fully defined by a single chemical structure. Each batch must be carefully characterized across multiple parameters, such as size distribution, surface markers, and cargo composition. Ensuring consistent quality therefore requires highly controlled sourcing, standardized production methods, and advanced analytical tools that extend beyond what is used for conventional drugs.

In your experience, what are the biggest challenges of working to encapsulate siRNA cargo into exosomes? Why are exosomes so well suited for carrying nucleic acid cargo?

Stability is the first challenge. The siRNA must be incorporated into the exosomes in a way that preserves both the integrity of the vesicle and the functionality of the RNA. Efficient loading is essential, but it cannot come at the expense of damaging or weakening the exosome structure. The goal is to achieve high loading efficiency while keeping the siRNA biologically available once delivered to the target tissue.

Exosomes are well suited for carrying nucleic acids because their natural lipid bilayer protects sensitive cargo and enables passage across biological barriers, including the blood–brain barrier. As nanoscale biological carriers, they can be administered in a minimally invasive way and combine two therapeutic functions: reducing inflammation and promoting tissue regeneration.

What types of investments are necessary to strengthen the manufacturing process for an exosome-based therapeutic company? What considerations did you have to make when selecting your master cell bank to create high quality exosome-based therapies?

We have made significant investments in infrastructure. We acquired a master cell bank, securing a reliable source of critical raw materials and strengthening its manufacturing process and supply chain in preparation for upcoming clinical studies and future patient treatments. Our production process is fully three-dimensional and designed for large-scale GMP manufacturing.

Key investments focus on scalable bioreactor systems and GMP-compliant production facilities. Unlike cell therapy manufacturing, which requires very large bioreactors, our proprietary process allows high yields of exosomes from relatively small reactors. This means we can scale production efficiently without building massive infrastructure, achieving higher output with a smaller footprint.

We are also investing in technologies to further enhance the already stable shelf life of exosomes. Unlike cell-based systems, exosomes are acellular, do not proliferate or mutate, and are inherently more stable. Through optimized formulation and storage, we can extend this advantage even further, supporting large-scale distribution and long-term global supply readiness.

To help our readers understand and potentially grow their own exosome-based businesses, can you elaborate on NurExone’s unique approach?

Our approach is multi-pronged. For starters, we use a targeted CNS focus. Unlike many biotechnology companies pursuing broad therapeutic applications, NurExone has focused its efforts on CNS-specific treatments, allowing for deeper specialization and expertise in this complex therapeutic area.

Secondly, we believe treatment can, and should be, minimally invasive. Our delivery system offers patients a non-surgical treatment option that could dramatically improve quality of life and accessibility to treatment.

We have extended our spinal cord injury therapy portfolio with new RNA sequences promoting healing and regeneration. In preclinical studies, we have demonstrated restored motor and sensory function in models of injury where healing and self-repair would not naturally occur.

In addition to spinal cord trauma, we have focused our research on dual indication potential. Using exosomes, we have also demonstrated promising results in optic nerve damage repair, suggesting potential applications for optic nerve treatment.

How do you plan to expand on these successes and what can NurExone’s experiences offer the overall RNA community?

We envision a day, in the not-too-distant future, when an American football player or traffic accident victim is treated for a spinal cord injury by paramedics at the scene with a minimally invasive treatment. Maybe it won’t even be necessary to go to the hospital.

For the RNA therapeutics community, our approach demonstrates the potential for exosome-delivered, RNA-based treatments to treat patients with CNS injuries, offering hope where traditional therapies have fallen short.

About the Expert:

Lior Shaltiel, Ph.D., is an entrepreneur and an award-winning scientist with extensive multidisciplinary international experience, specializing in chemical engineering, molecular biology, electrophysiology, pharmacology, and drug delivery systems. Lior has years of experience in accelerating Israeli start-ups. He has worked for several nano-drug delivery companies such as LipoCure and Ayana Pharma. Before joining NurExone, Lior was a VP and Partner at a boutique Chinese investment bank operating in Israel, mapping the investment landscape and opportunities in the Israeli pharmaceutical industry. Lior is the initiator and head of the BioMed-MBA program at the Hebrew University.