Guest article by Gregg Draudt- Head of US Innovation Strategy & Business Development at Fearsome

At PODD24, AAO and other drug delivery and diagnostics focused conferences this past fall, Neuro and Ocular therapies & delivery systems were highlighted as the tip of the spear in terms of innovation. I thought it was worth a bit of a deeper dive to understand just why that is the case. It was so very relevant to us at Fearsome - as these markets make up ~80 % of our client work over the past five years.Â
Neuro and ocular therapies are considered cutting-edge in gene and cell therapies due to the unique challenges and immense potential they offer for treating complex, often untreatable, conditions. Here are the key reasons these anatomical targets are on the forefront of drug delivery innovation.
Unmet Clinical Needs
Many neurodegenerative disorders (e.g., Alzheimer’s, Parkinson’s, ALS, Huntington's disease) currently lack curative treatments. Traditional treatments often fail to address the underlying cause of these diseases, as they cannot cross the blood-brain barrier or provide targeted, long-term solutions. Ocular conditions like retinitis pigmentosa, macular degeneration, and glaucoma lead to irreversible vision loss and often have limited treatment options. Gene and cell therapies offer a way to address genetic and cellular causes of these diseases, rather than just managing symptoms.
Targeted Delivery & Dose Size
Delivery to the brain requires bypassing the blood-brain barrier, a challenge in need of new levels of precision. Techniques like direct intraparenchymal injections, intracerebroventricular delivery, ultrasound mediated delivery, brain shuttle, and convection-enhanced delivery (CED) have begun to allow for targeted, localized treatment with novel methods of dose control. Recently, for instance, Neurochase has been developing methods to optimize CED infusion performance - gaining an understanding of the interface between device and tissue, and enhancing the predictability of drug distribution in the brain. This is a critical factor for the safe, effective and scalable delivery of many emerging brain targeted pharmaceutical therapies. Scalability is important as existing solutions are resource heavy and quite invasive - taking up to 18 hours with a patient under GA in an MRI scanner. The eye’s compartmentalized structure (e.g. subretinal and intravitreal spaces) enables precise, localized delivery of therapies directly to the affected tissues, minimizing systemic exposure and potential side effects. This makes ocular conditions particularly suited to gene therapies.
Immune Privilege -Â Lower Risk -Â Faster Market Impact
As an extension of the central nervous system, the eyes share an anatomical connection with the brain. Besides the visual connection, both the brain and the eyes are considered immune-privileged sites, wherein the immune response to pathogens is highly controlled to preserve the organ’s functions and minimize potential damage to tissues that do not easily regenerate. In other words - ‘immune privilege’ means that the immune system’s response is limited within these regions - which lowers the risk of immune reactions and rejection of cell-based therapies or viral vectors delivering gene therapies, increasing the likelihood of successful integration and function.
Genetic basis & the Promise of Long-term Benefit
Many neurological disorders have a genetic basis (e.g., Huntington’s, spinal muscular atrophy, ALS and more). Gene therapy can directly address these genetic mutations, offering the potential for lasting effects and modifying disease progression. A large proportion of retinal diseases are caused by a mutation in a single gene (monogenic), making them prime candidates for gene therapy - and by replacing or repairing faulty genes, gene therapies can restore or preserve vision for long periods.
Advancements in Science and Technology
Advances in viral vectors (such as AAVs, or adeno-associated viruses) have improved delivery mechanisms for brain-targeted therapies. Technologies like CRISPR, ASOs (antisense oligonucleotides), and RNA interference are showing promise for precise genetic correction or suppression within the central nervous system.Specific to the eye - its small size and isolated nature allow for early adoption of cutting-edge gene-editing technologies. Therapies using CRISPR, optogenetics (for restoring light sensitivity to damaged retinal cells), and stem cells are progressing in clinical trials for eye diseases.
Regulatory Support and Advancing Clinical Trials
Regulatory agencies, including the FDA, have been creating pathways for accelerating the development and approval of gene and cell therapies, especially for rare and debilitating diseases. Fast-track designations, orphan drug status, and regenerative medicine advanced therapy (RMAT) designations help expedite approval and attract investment. Luxturna (for RPE65-related retinal dystrophy), has set a precedent, encouraging further investment and development in neuro and ocular gene therapies.
Investment and Interest from Biotech and Pharma
Given the aging population and the increasing prevalence of neurodegenerative diseases, pharmaceutical and biotech companies are heavily investing in innovative approaches like gene therapy. These therapies promise long-term, potentially curative effects compared to traditional drugs that primarily treat symptoms.The eye is one of the first areas where gene therapy has shown success, with several high-profile successes spurring additional investment. The relatively lower risk and shorter development timelines for ocular therapies make them attractive to investors. Access through the skull is a bigger challenge than access within the eye but new delivery technologies are showing great promise.
Precision Medicine Potential and Patient Impact
Neuro and ocular therapies align with the trend toward precision medicine, as treatments are often tailored to individual genetic mutations. For example, gene therapies can be designed for specific mutations that cause conditions like spinal muscular atrophy or Leber congenital amaurosis. For patients facing blindness or severe visual impairment, gene and cell therapies offer hope for functional improvement or even restoration of sight, dramatically improving quality of life.
In Conclusion
The answer to why there has been such a focus on Neuro & Ocular is because, within them, therapies can address significant unmet needs with potentially transformative outcomes. Pharma and MedTech developers are able to leverage advanced delivery technologies in immune-privileged environments, and benefit from regulatory support and investment. These targets and therapies are poised to create paradigm shifts in treating conditions that were previously considered incurable - and new systems for delivery will require extraordinary inventiveness, precision and finesse to enable surgeons to provide them for patients.
If you would like to continue this discussion, or learn more about how Fearsome might help your team refine or create new to the world delivery systems, please reach out to Gregg at gregg.draudt@fearsome.co.uk.
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