Key Considerations of Gene Therapy Development: A Caregiver's Perspective

Emily Walsh Martin, PhD - June 22, 2022

Check back to our blog every week to learn more about Dr. Walsh Martin's journey and the emerging gene therapy efforts in syndromic and monogenic epilepsies.

For this blog post, I wanted to get a caregiver’s perspective on these therapies who could talk about their promise and the unknowns. As someone who helps drug development teams navigate the unknowns prior to first-in-human testing of a novel therapy, I wanted to understand how our teams can be better partners to the caregivers in the patient’s life. Ultimately, the particulars of a gene’s function and “correctability” probably play an outsized role in what level of efficacy can be achieved for a patient. But certainly, there are things we can build into the development efforts and our communication with caregivers that can have meaningful impact. 

Moreover, as someone who has seen teams struggle with the selection of doses for clinical study, which was discussed in the last blog post, I wanted to understand how a caregiver thinks about the right time to join a trial, and how they weigh the concerns of treating their loved one as early as possible against the risk of joining a trial at the first dosing level when there are often many more unknowns about the therapy’s efficacy and toxicity. I spoke with Amber Freed, founder and CEO of SLC6A1 Connect, a patient organization focused on accelerating the development of therapies for SLC6A1 patients like her son Maxwell.

Read more about Amber's story on our blog.

Emily Walsh Martin is a volunteer crew member for Sail For Epilepsy’s Atlantic crossing on the vessel Ingwe. When she’s not sailing, she is a consultant for gene and cell therapy companies and investors who are seeking to advance novel therapies in the clinic.