UniCure Reports Positive 3-Year Huntington’s Gene Therapy Data: Unique IP Considerations for Complex Gene Therapy Programs

UniCure announced positive 3-year data for their Huntington’s disease gene therapy this week, with the company receiving FDA breakthrough therapy designation and planning to file for approval in Q1 2026. The treatment uses stereotactic brain surgery to deliver gene therapy directly to the striatum, where it silences production of the mutant huntingtin protein that drives this autosomal dominant neurodegenerative disease.

Several unique aspects of this program raise distinctive IP considerations for companies potentially entering similar regulatory and development paths.

External Control Studies Create Supplemental IP Opportunities:

UniCure received FDA breakthrough therapy designation based on comparing treated patients against natural history controls rather than traditional placebo-controlled trials. While this doesn’t fundamentally change the standard method of treatment claims, it does create opportunities for additional patent strategies that supplement traditional approaches.

Companies can still pursue conventional methods of treatment claims for treating Huntington’s disease, but external control pathways open up supplemental claim strategies around the specific methodologies that enable this regulatory route. Patent applications might include method claims covering the statistical analysis protocols used to validate external controls, patient selection algorithms that ensure appropriate matching to natural history cohorts, and disease progression modeling techniques that account for inter-patient variability in longitudinal comparisons.

These supplemental claims create additional layers of IP protection around the clinical development and regulatory strategy itself. While competitors might design around traditional treatment method claims, they would still need to navigate the specific external control methodologies if they want to pursue similar regulatory pathways. This is particularly valuable in rare diseases where the external control approach may become the preferred or only feasible development path for follow-on therapies.

This regulatory pathway creates unique patent strategy considerations.

Delivery Method Patents Become Critical Differentiators

UniCure’s stereotactic brain delivery isn’t just a medical necessity—it’s potentially a strong IP moat. Unlike systemic delivery methods that can’t effectively cross the blood-brain barrier to reach the striatum, stereotactic delivery allows precise targeting of the exact brain regions where huntingtin pathology occurs. When you can’t rely on head-to-head efficacy comparisons against competitors, your delivery mechanism, surgical protocols, and administration techniques become key competitive advantages. Patent applications should comprehensively cover the specific brain coordinates and injection trajectories that optimize therapeutic delivery to the striatum, along with catheter designs specifically engineered for brain tissue penetration without causing damage. Multi-site injection protocols and their sequencing become particularly valuable IP, as does the real-time monitoring technology used during stereotactic procedures to ensure accurate placement and delivery.

Biomarker Strategy Must Evolve

Traditional pharmacodynamic markers, such as cerebrospinal fluid (CSF) huntingtin protein levels, may not correlate with therapeutic activity in the deep brain regions where the gene therapy is actually effective. This creates a disconnect that forces a broader consideration of what constitutes measurable innovation. The challenge is that CSF measurements might not accurately reflect what’s happening in the striatum, where the therapy is delivered, making traditional biomarker approaches less reliable. Alternative monitoring approaches specific to stereotactic delivery, clinical assessment methods that support external control comparisons, and patient selection algorithms that can reliably match natural history cohorts all become patentable subject matter. Disease progression modeling techniques that enable meaningful comparisons to historical data represent another layer of potentially valuable intellectual property in this regulatory environment.

Regulatory Exclusivity Considerations

With Huntington’s affecting roughly 30,000 patients in the US, UniCure likely qualifies for 7-year orphan drug exclusivity, which provides market protection that runs concurrently with patent protection rather than replacing it. This creates complementary layers of exclusivity that must be considered together in patent strategy. The orphan exclusivity begins at FDA approval and provides market protection regardless of patent status, while patents protect the underlying invention for their full term.

The strategic consideration is to ensure that patent claims align with and reinforce the regulatory exclusivity period. Since orphan exclusivity prevents competitors from getting FDA approval for the same indication, the patent strategy can focus on protecting against potential design-arounds or alternative approaches that might not trigger the orphan exclusivity protection. This might favor broader method claims regarding the general therapeutic approach, while also including specific claims about the unique delivery methods and patient populations that enabled the external control regulatory pathway.

As FDA shows increasing flexibility in rare neurodegenerative diseases, patent prosecutors must think beyond traditional claim strategies. The question becomes whether we’re adequately protecting the unique innovations that make non-traditional regulatory pathways possible, and how patent strategy should evolve to reinforce rather than merely supplement regulatory advantages.