CMTA names Johns Hopkins’ Brendan Zotter as 2027 fellowship recipient

The Charcot-Marie-Tooth Association has selected Johns Hopkins clinician-scientist Brendan Zotter, MD, PhD, for its 2027 Inherited Neuropathies Fellowship through the CMTA-INC Alliance. His project focuses on improving gene therapy delivery for Charcot-Marie-Tooth disease, a rare inherited nerve disorder with no approved treatment.

Why it matters: - The fellowship is aimed at building the next generation of CMT researchers and clinician-scientists. - Zotter’s project targets gene delivery, a key hurdle for turning genetic therapies into treatments for people with Charcot-Marie-Tooth disease. - CMT remains an inherited nerve disorder with no approved treatment.

What happened: - The Charcot-Marie-Tooth Association announced Johns Hopkins physician-scientist Brendan Zotter, MD, PhD, as the 2027 Inherited Neuropathies Fellow. - The award comes through the CMTA-INC Alliance Inherited Neuropathies Fellowship. - Zotter will use the fellowship to study better ways to deliver gene therapy for CMT.

The details: - Zotter will spend the next year developing and testing improved adeno-associated virus, or AAV, vectors. - The vectors are designed to deliver genetic therapies more efficiently to Schwann cells. - Schwann cells produce and maintain the myelin that protects peripheral nerves. - Zotter will test the vectors in a CMT mouse model. - The work is intended to improve gene delivery approaches that could one day help people with multiple types of CMT. - Zotter earned his medical degree and doctorate at New York University. - Zotter completed his neurology residency at the University of Pennsylvania. - His prior research at Children’s Hospital of Philadelphia focused on viral vectors that target Schwann cells for genetic therapy. - At Johns Hopkins, Zotter is continuing that work under the mentorship of Charlotte Sumner, MD, while completing a clinical fellowship in neuromuscular medicine.

Between the lines: - The fellowship is designed to give early-career physician-researchers protected research time, mentorship, and CMT-focused clinical exposure. - That support can help move promising lab work toward independent research programs. - Mary M. Reilly, MBE, MD, said developing clinician-scientists is essential to advancing care and research in CMT. - Reilly also said the program provides structured support to help early-career physicians build the expertise needed for independent careers and future CMT research. - Zotter said the fellowship will give him focused experience in CMT translational research and mentorship for building an independent program developing genetic therapies for CMT.

What's next: - Zotter will carry out the fellowship work over the next year. - The fellowship will continue to support early-career clinician-investigators with research time, long-term mentorship, and career development opportunities. - CMTA said the program has supported 11 fellows since its inception, with alumni contributing to CMT research and clinical progress worldwide. - Readers can find more information at cmtausa.org.