A Sponsor's Blueprint For Maintaining Compliance And Ethics In Gene Therapy Trials For Neurological Conditions

By Thos Cochrane, Director, Fellowships in Bioethics, Harvard Medical School; Linda Reuter, Senior IRB Director, BRANY; Raffaella Hart, Senior Vice President, IRB and IBC Services, BRANY; and Vanessa Rodriguez, IRB/IBC Supervisor, BRANY

In the pursuit of creating effective gene therapies for treating neurological conditions — both neurodevelopmental and neurodegenerative — researchers must navigate the complexity of how these diseases manifest clinically. Often, these studies involve vulnerable populations, either those with cognitive impairment or pediatric participants, subjecting them to distinct ethical, regulatory, and biosafety challenges. As a result, sponsors must think critically about how to ethically approach informed consent.

To create efficacious treatments for these conditions, drug sponsors must design safe and ethical clinical trials that protect participants and their caregivers while maintaining compliance with regulators. This includes designing a nuanced informed consent process, planning for a surrogate consent provider in the instance that a participant loses capacity, and anticipating risks for all involved. Download the full article for a deeper look at the IRB and IBC review process as well as expert insight into building an ethical approach to informed consent.