Dr. Meghan Drummond, Vice President of Research and Drug Development at the CMT Research Foundation
The Food and Drug Administration recently approved Otarmeni, a gene therapy developed by Regeneron. The program was spearheaded by Meghan Drummond, PhD, who now serves as the Vice President of Research and Drug Development at the CMT Research Foundation.
The newly-approved treatment restores hearing in children born deaf due to otoferlin deafness, a rare inherited condition called auditory neuropathy, caused by a mutation in a single gene, similar in some ways to many types of CMT.
“Children born deaf face a neurodevelopmental emergency,” Drummond explains. “There is a window of time where becoming familiar with language is crucial, and listening is a powerful tool in that stage. That’s why we needed to intervene as early as possible. We are giving these children some of their most powerful learning opportunities.”
Dr. Drummond led the scientific strategy and execution that brought this therapy from concept to approval. Now, she’s leading scientific strategy once again—this time, targeting Charcot-Marie-Tooth disease.
The parallels to CMT are meaningful. For example, many kinds of CMT are similarly driven by well-characterized genetic mutations, and like otoferlin deafness, it presents researchers with defined biological targets. The challenge in both cases is translating that genetic understanding into a safe and effective therapeutic strategy, exactly the kind of work this FDA approval proves Dr. Drummond can lead at the highest level.
Dr. Drummond’s experience spans the full arc of rare disease drug development, from pre-clinical models through IND approval, clinical execution and regulatory approval. That breadth of expertise is precisely what CMT research needs as the field moves closer to viable treatments. Rare diseases require champions who understand both the science and the strategic pathway to get therapies to patients, and Dr. Drummond has proven she is that champion.
The approval of Otarmeni is a reminder that even the most daunting rare disease challenges can be solved with the right scientific vision, rigorous execution, and sustained commitment. Dr. Drummond brings all of that to CMTRF, and we look forward to the progress ahead.
Read more about Otarmeni in The New York Times or hear from a patient and his mother on CNN.
CMTRF is a science-first organization, with an aggressive research agenda led by Dr. Drummond. You can help empower Dr. Drummond’s leadership at CMTRF with a donation.
You can hear from Dr. Drummond directly in our Gene Therapies Demystified webinar series.