Stanford Student Wins Fellowship to Engineer Her Own Cure

Diagnosed with facioscapulohumeral muscular dystrophy at age 13, bioengineering student Héloïse Hoffmann has secured an O'Shaughnessy Fellowship to advance a novel gene therapy. The Stanford researcher is now moving her project from the laboratory to animal models, aiming to neutralize the toxic protein responsible for the rare muscle disease.

Hoffmann’s research at the Stanley Qi Lab focuses on blocking DUX4, a protein that triggers muscle-wasting gene activity. While traditional efforts attempt to suppress DUX4 production entirely, her method utilizes an engineered protein to prevent the toxin from activating harmful pathways. Her preliminary results in patient cells show the technique effectively mitigates DUX4 activity. She now plans to validate these findings in mouse models, establishing a potential framework for treating other conditions caused by similar gene dysregulation.

Her path to this fellowship includes a gold medal at the 2024 International Genetically Engineered Machine competition and a $50,000 grant from the Sarafan ChEM-H Undergraduate Entrepreneurship Program. Beyond the lab, she founded GenZ of FSHD to mobilize patient advocacy and previously refined her expertise through an internship at Regeneron Pharmaceuticals. Jim O'Shaughnessy, CEO of O'Shaughnessy Ventures, emphasized that Hoffmann’s combination of scientific rigor and personal stakes makes her an ideal candidate for the program, which provides up to $100,000 in funding and access to an expert network.