Sano recently worked with a clinical-stage biotech advancing a gene therapy program for a rare cause of congenital hearing loss — a context that illustrates several of the structural challenges common to genetically stratified rare disease trials at the Phase 2/3 boundary.
Traditional recruitment channels were insufficient for reaching the small pool of genetically confirmed patients. In genetically stratified trials, eligibility cannot be determined by clinical diagnosis alone. Patients must first undergo genetic testing, and many potential participants had never been tested. Families often encountered logistical or emotional barriers to pursuing newborn genetic screening.
The sponsor needed a partner who could not only identify and engage these families, but also do so in a way that respected community perspectives and reduced barriers between genetic testing and trial enrollment.
Key Takeaways
- High Engagement: Patient education open and click rates more than doubled industry averages.
- Effective Screening: 510 individuals completed the pre-screener with an 85% eligibility rate.
- Streamlined Funnel: 20% of pre-screened participants were referred directly to genetic testing or the active study.
- Scalable Model: The regulatory-ready platform is now expanding the trial into the UK market.
How patients were identified and kept engaged
Sano developed and implemented a tailored patient-finding protocol for this study, designed to engage both participants and healthcare providers through a range of awareness and education channels. This included consent flows, recruitment materials, a study questionnaire, and structured communications including emails, alerts, and notifications.
Because patients and caregivers learn about clinical research through a wide range of sources, from their physicians to online search, Sano deployed an omni-channel recruitment approach spanning:
- In-person events
- Point-of-care engagement with audiologists, pharmacists, nurses, and early hearing diagnosis specialists
- Digital advertising
- Partnerships with hearing loss advocacy groups
- Social media and website content
- Provider-facing materials
All channels directed interested individuals to a dedicated study page that served as a central hub for education, FAQs, and the screening questionnaire.
Caregivers of potential participants with prior genetic testing results could securely upload them through the platform, while those without existing results were referred to a genetic testing partner. Uploaded reports were used to confirm self-reported diagnoses.
Once genetic analysis was complete, eligible individuals were segmented into one of two study cohorts. Those who did not immediately qualify were enrolled in the Sano Virtual Waiting Room — a compliant engagement environment that maintains an active relationship with genetically confirmed participants, enabling recontact for future studies without rebuilding identification infrastructure from scratch. This approach streamlined the path from awareness to enrollment while preserving engagement for individuals who may qualify for future studies.
Clear, consistent communication was central to the program's performance. Open and click rates for patient education content and newsletters more than doubled industry averages. This sustained connection helped keep participants informed and engaged through each step of the process.
Study outcomes
Overall, 510 individuals completed the pre-screener, demonstrating the reach of the patient-finding protocol in a population where genetically confirmed patients are rare. Of these, 85% were found to be eligible, and 20% were referred to genetic testing or to the current study.
In an environment where most trials struggle to meet enrollment targets on time, these conversion rates reflect the value of a coordinated approach that integrates recruitment, genetic testing, and patient education within a single workflow.
Building on these results, the study will soon launch in the UK with Sano managing the patient-finding, pre-screening, and referral process in a regulatory-ready format. Because the platform, workflows, and engagement infrastructure are already in place, the sponsor can expand to a new geography without rebuilding operational processes from scratch. This reduces the timeline and coordination burden typically associated with multi-country rollouts.
Conclusion
These results demonstrate that coordinated recruitment, culturally sensitive engagement, and a regulatory-ready platform can meaningfully accelerate enrollment in genetically defined trials. The approach Sano deployed for this program is designed to be replicated across regions and adapted to other rare disease populations.
Realizing the full potential of gene therapy depends on solving a structural problem: connecting genetically eligible patients to trials before enrollment windows close. In rare disease, where patient populations are small and site-level under-enrollment is common, fragmented workflows introduce compounding risk at every step.
By integrating recruitment, genetic testing, patient education, and long-term engagement within a single system, Sano reduces the operational gaps that delay enrollment and limit access. The result is not just faster trials, but more reliable ones.