Accelerating a gene therapy trial with Sano’s support

In recent years, the therapeutic landscape for genetic hearing loss (GHL) has advanced rapidly, with several programs reaching clinical development. However, trials in this space can present unique challenges. Recruiting patients often requires careful engagement with the deaf and hard-of-hearing community, where cultural and linguistic considerations are paramount. The rarity of certain genetic variants can make it difficult to recruit patients who meet inclusion criteria. In addition, newborn genetic screening is a critical pathway for early diagnosis, but it may not always be available or welcomed by parents. 

Sano recently worked with a biotech struggling with these hurdles firsthand when preparing a trial for a gene therapy targeting a rare cause of congenital hearing loss. Traditional recruitment channels were insufficient for reaching the small pool of genetically confirmed patients. Many potential participants had never been tested, and 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 accelerated the path from genetic testing to trial enrollment.

Sano’s approach to finding patients and keeping them engaged

To achieve success for our client, we developed and implemented a tailored, patient-finding protocol for this study, designed to engage both participants and healthcare providers through a variety of awareness and education channels. This included consent flows, recruitment materials, a study questionnaire, and a suite of communication touchpoints (emails, alerts, notifications). 

Recruitment outreach spanned in-person events, point-of-care engagement with audiologists, pharmacists, nurses, and early hearing diagnosis specialists, digital ads, and partnerships with hearing loss advocacy groups. Social media, website content, and provider-facing materials directed interested individuals to a dedicated study page that acted as a central hub for education, FAQs, and the screening questionnaire.

Caregivers of potential participants with prior genetic testing results available could securely upload results, while those without 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 or placed in the Sano Virtual Waiting Room, which is a secure digital portal offering tailored research opportunities and educational content. This integrated approach streamlined the path from awareness to enrollment, while maintaining engagement for those who may be eligible for future studies.

Our focus on clear, consistent communication drove exceptional engagement: open and click rates for patient education and newsletters more than doubled industry averages. This level of sustained connection helped keep participants informed and motivated to complete each step.

Study outcomes

Overall, 510 individuals completed the pre-screener, which highlights the effectiveness of our patient-finding protocol for this study. Of these, 85% were found to be eligible, and 20% were referred to genetic testing or to the current study.

Building on this success, the study will soon launch in the UK with Sano managing the patient-finding, pre-screening, and referral process in a regulatory-ready format. By relying on our streamlined processes, sponsors can scale to new regions without additional infrastructure burden, accelerating timelines and making expansion much simpler.

Conclusion

These results are highly promising and have enabled the sponsor to move closer to their goal of bringing a groundbreaking gene therapy to patients who need it. By combining precision recruitment, culturally sensitive engagement, and a regulatory-ready platform, Sano delivered a scalable solution that can be replicated across regions.

Realizing the full potential of gene therapy depends on finding the right patients quickly, running efficient trials, and delivering treatments at scale. By removing operational and logistical barriers, Sano helps ensure these therapies reach patients faster and with greater impact.