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CASE STUDY: GENE THERAPY FOR RARE DISEASES

Advancing Rare Disease Clinical Trials with Point-of-Need Eye Assessments

THE CHALLENGE

Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company, faced significant challenges during the COVID-19 pandemic and subsequent SKYLINE trial enrollment for X-linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM) gene therapy treatments. Travel restrictions and site limitations hindered patient participation and threatened trial progress.

20/20 ONSITE SOLUTION

20/20 Onsite partnered with AGTC to deploy Mobile Vision Clinics (MVCs) equipped with state-of-the-art ophthalmic equipment, including OPA, MAIA, AVOT, OCT, and CoBri tests. These MVCs traveled directly to patients' homes, providing a safe, convenient, and efficient environment for conducting crucial assessments and pre-screenings.

mobile vision clinic parked on the road
eye assessment being performed on patient

RESULTS

PANDEMIC RESCUE:

AGTC successfully reported ophthalmic data from all ongoing trials, completing 150 assessments across 24 states and 4 trials.

HIGH PATIENT SATISFACTION:

97% of participants recommended the service, praising the convenience, comfort, and safety of the MVC experience.

ENROLLMENT SUCCESS:

Exceeded enrollment goals with 14 participants, utilizing MVCs for pre-screening and rescreening.

COSTLY DELAYS AVOIDED:

Avoided costly trial delays and participant dropout associated with traditional assessment methods.

97%
Participants Recommended
150+
Assessments Completed
24
States with Patients

FINAL ASSESSMENT

20/20 Onsite's eye assessment solution proved instrumental in enabling AGTC to navigate its specific challenges and achieve its clinical trial goals. By prioritizing patient needs and delivering high quality, accessible care, we helped AGTC accelerate progress toward developing treatments for XLRP and ACHM.

Partner with 20/20 Onsite to overcome your clinical research challenges and bring eye assessments directly to your enrollees. Schedule a consultation and explore how our point-of-need solutions can support your clinical research.

"We recognize the incredible commitment patients make when they enroll in our trials and our goal is to match that commitment and support them in any way possible. By using a creative patient-focused approach to testing via 20/20 Onsite's mobile vision testing program, we take the test to the patient to support their needs during this extremely difficult time."

Sue Washer
-Sue Washer, CEO, AGTC

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