Today, we sit down with Dr. Lior Shaltiel of NurExone Biologics to explore how exosome-based delivery of siRNA could redefine CNS repair. We discuss why exosomes might overcome safety and manufacturing hurdles that hampered cell therapies, how early preclinical signals are guiding a rigorous first-in-human path, how endpoints and patient realities shape trial design, and the broader potential of exotherapy as a platform for regenerative medicine. This conversation highlights leadership in translating promising science into a practical regulatory and business strategy amid a challenging funding climate.
Moe: I’m curious why you chose exosome delivery for CNS injuries over cell therapies, given safety, BBB crossing, and manufacturing hurdles seen in prior regenerative efforts.
Dr. Lior Shaltiel: I chose exosomes as a delivery mechanism because they are natural, nonliving carriers with inherent healing properties that can access injured CNS tissue with minimal invasiveness and a lower safety risk than living cells. They can also deliver a therapeutic payload directly to sites of inflammation. After CNS injury, inflammation gates regeneration; exosomes target inflamed tissue, cross the blood–brain barrier, and deliver a dual-action payload that first dampens harmful inflammation then promotes neural repair. Our technology for loading therapeutic payloads into exosomes after production transforms this into a platform, not a one-off development.
This production and loading process gives us precise control over both the exosome production and therapeutic payload, avoiding the complexity of engineering cells. The cell-free approach supports scalability, batch-to-batch consistency, and rapid adaptation to new targets. By defining the exosome and its payload as the drug, we simplify safety profiling and regulatory dialogue while enabling expansion into additional indications that share the same inflammatory-regenerative biology. This aligns with our strategy to start with our lead candidate ExoPTEN, for spinal cord injury and extend to optic nerve damage, traumatic brain injury, and stroke as the biology allows.
The translational advantage is clear: exosomes reduce safety burdens, enable a cleaner manufacturing path, and provide a versatile carrier for payloads. If we keep a tight process including proven exosome-loading post-production, and robust data demonstrating regeneration and functional gains, we have a credible path to IND that regulators can review against well-defined endpoints and an adaptable and versatile platform for future indications. The platform mindset also invites collaboration with pharma for novel payloads for other indications, accelerating development while maintaining safety and reproducibility as core values.
Moe: What early signals from your preclinical work will you emphasize in the first-in-human design to avoid the valley of death and satisfy regulators?
Dr. Lior Shaltiel: The clinical plan will be centered on observable biology with regulatory credibility. Our preclinical work with ExoPTEN shows neurons regenerating, injury gaps closing, and functional recovery in rats treated with high-dose, with many regaining the ability to walk. We’ve engaged the FDA in pre-IND discussions and are moving toward a formal IND, with clear CMC, a robust preclinical package, and a clinical-trial synopsis. In humans, I’ll stress multi-modal readouts: histological regeneration, motor and sensory improvements, bladder function, and patient-centric quality-of-life proxies. The design is expected to target a two-week treatment window with two to four injections, balancing timely recovery signals with patient burden. Importantly, we’ll present a dose-optimized strategy anchored in reproducible biology, reinforcing the translational credibility that regulators require and that patients deserve.
We’re also building a data package that demonstrates dose-response consistency and a clear mechanism—inflammation reduction followed by regeneration—so investigators and regulators can forecast meaningful outcomes. By aligning the trial design with tangible, measurable endpoints and the rehabilitation arc that follows, we create a credible narrative of incremental, real-world benefit. This approach helps bridge the gap from compelling animal data to human safety and efficacy signals, reducing uncertainty and facilitating a smoother IND review, while preserving the scientific rigor needed for confident decisions by regulators and clinicians alike.
Moe: How will you define Asia A to Asia B endpoints and patient selection for acute SCI, balancing heterogeneity, rehab potential, and meaningful function gains?
Dr. Lior Shaltiel: Our focus is on acute SCI because chronic injury carries extensive degeneration and scarring that limit recovery. The primary endpoint is the transition from ASIA A to ASIA B—gaining control over the most affected functions. This aligns with patient priorities: motor and reflex gains enabling rehabilitation and daily living improvements. We’re pursuing a two-week window with two to four injections to maximize the chance of a detectable functional shift within a practical period. We’ll support these endpoints with objective measures across motor and sensory function, bladder control, and where feasible, quality-of-life indices. By selecting the most severe cases, we target a population where even modest gains translate to meaningful clinical change, while rehabilitation can amplify long-term recovery.
To account for heterogeneity, we’ll stratify by injury mechanism and level, apply standardized assessment timelines, and include rehabilitation outcomes as a secondary, yet critical, readout. This design supports a robust signal for future development without overpromising, and it respects the real-world trajectory patients and caregivers experience as they pursue recovery within a constrained treatment window.
Moe: How will you manage expectations and communicate progress honestly, without inflating narratives that could mislead patients, regulators, or investors?
Dr. Lior Shaltiel: Our communications are anchored in rigorous science and reproducible results. We tested our approach in stringent models, including full spinal cord transection, to prove that regeneration and functional recovery are possible under challenging conditions. We then refined exosome sourcing and loading for consistent potency. We emphasize a platform narrative—Exotherapy—over a single indication, and I’ll lay out realistic timelines: IND filing next year, incremental data releases, and the role of rehabilitation in achieving longer-term gains. By presenting incremental, verifiable milestones—functional improvements—I aim to sustain trust with patients, clinicians, regulators, and investors. We’ll acknowledge uncertainties and avoid overpromising, ensuring transparency about the healing trajectory and the need for continued monitoring and combined therapies to maximize outcomes.
I also plan to share detailed, peer-aligned data packages and update letters with regulators to support regulatory confidence, while engaging patients and advocacy groups with clear explanations of what may or may not be achievable within specific timeframes. The balance between ambition and restraint is essential to maintain credibility as we progress through IND and beyond.
Moe: Can you point to a regulatory or manufacturing uncertainty that forced you to rethink the approach, and what does that reveal about the field’s maturity?
Dr. Lior Shaltiel: Early exosome work relied on engineered exosomes from modified cells, which introduced safety, manufacturing, and regulatory hurdles. We pivoted to a cell-free paradigm where exosomes themselves are the actual drug. In our case, they are loaded with therapeutic molecules in post-production. This move improves process control, scalability, and collaboration potential with pharma partners, reducing dependence on a single cell source and its associated risks. It also clarifies the regulatory path by focusing on the exosome as the drug and the payload as a defined, controllable component, enabling better characterization and comparability across batches. This shift mirrors a broader industry lesson: define the therapeutic platform clearly, maintain strict quality controls, and design products that regulators can review with consistent, transferable data. It’s about turning promising biology into a robust, deliverable pipeline.
The field’s maturity is reflected in the now widely adopted “exosome as the drug” mindset, the emphasis on post-production loading, and the push toward modular partnerships that expand payload variety without reengineering the entire production system. By reducing cellular variables and building a reproducible, scalable framework, we’re setting a practical standard for how exosome therapies can progress through regulatory and manufacturing realities toward real patients.
Moe: Looking ahead, how do you see Exotherapy as a platform shaping regenerative medicine—broad CNS indications or neuron-centric expansion—and what are the key levers?
Dr. Lior Shaltiel: I see Exotherapy as a neuron-centric platform with broad potential beyond SCI. The neuronal targeting of our exosomes suggests applicability to optic neuropathies, other CNS injuries, and certain neurodegenerative conditions, provided the payloads address specific disease mechanisms. The platform’s expansion hinges on payload versatility, exosome sourcing, targeting fidelity, and manufacturing scalability. Critical levers include maintaining neuron-selective uptake, refining loading strategies for diverse targets, and ensuring modular, shareable manufacturing processes so pharma partnerships can scale quickly. Demonstrating a modular, adaptable payload strategy will be essential to extending Exotherapy beyond its first indications while preserving safety, regulatory compliance, and data integrity. In short, a platform that can deliver multiple neuronal payloads via a consistent, well-characterized exosome system has the potential to redefine regenerative medicine across CNS and neuron-centric diseases.
This vision depends on proving consistent potency across exosome sources, establishing robust QC for post-production loading, and building a manufacturing infrastructure capable of rapid payload swaps. If we can couple strong preclinical signals with a clear regulatory pathway and collaborative development models, Exotherapy could become a versatile tool for neuron-driven recovery, not just a spinal cord breakthrough.
Moe: From a business and funding perspective, you’ve pursued a public-market path through difficult biotech cycles. What lessons stand out about sustaining late preclinical programs, and what guidance would you offer on capital strategy and risk management?
Dr. Lior Shaltiel: A public-path approach allowed us to weather a harsh funding climate by preserving visibility, discipline, and a data-driven narrative. We’ve raised nearly US$18 million in preclinical funding and earned recognition as a 2025 Toronto Stock Exchange Venture top performer, with a patient, international investor base—especially German supporters who value long-term value. The lesson is that a platform strategy with measurable milestones, transparent data, and prudent capital allocation can offer an alternative to VC-heavy models. It helps to maintain liquidity, pursue strategic partnerships, and diversify funding channels beyond traditional bioscience VC.
For the field, I’d advise founders to articulate a credible near-term regulatory path while preserving long-term growth through partnerships and scalable manufacturing. Focus on robust preclinical signals that regulators care about, align with patient needs, and structure milestones that attract patient and strategic investors who seek durable value rather than quick exits. If we show consistent data, disciplined capital use, and authentic engagement with regulators and patients, late-stage programs can survive and even thrive in a tough environment, underscoring the viability of an exosome-based platform in regenerative medicine.
Moe Alsumidaie is Chief Editor of The Clinical Trial Vanguard. Moe holds decades of experience in the clinical trials industry. Moe also serves as Head of Research at CliniBiz and Chief Data Scientist at Annex Clinical Corporation.
