Heloise Hoffmann
United States
FSHD gene therapy
Developing a gene therapy to block the activity of DUX4, the toxic protein at the root of FSHD, in order to stop the disease in its tracks.
Biography
Heloise Hoffmann is a patient-scientist working to cure the disease she lives with, facioscapulohumeral muscular dystrophy (FSHD).
Diagnosed with FSHD at thirteen, she has turned personal urgency into scientific purpose and is building a novel gene therapy platform for aberrant transcription factor-related diseases.
Heloise is driven by a simple conviction: the people closest to a problem are often the ones best positioned to solve it.
In Heloise's words
“Receiving this Fellowship means getting to dedicate my life to do the most meaningful work I can imagine: trying to cure the disease I and nearly one million others worldwide live with. I could not be more grateful for Jim and the OSV team for their belief in this mission and the vision of a world free from FSHD.”
Award
- 2026
O'Shaughnessy Fellowship
Developing a gene therapy to block the activity of DUX4, the toxic protein at the root of FSHD, in order to stop the disease in its tracks.
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