Mapping leukodystrophy pipelines by disease biology

Although drug development remains limited for leukodystrophies, more than two dozen biotechs are now matching therapeutic modalities to the genes, pathways and cell types driving distinct forms of white matter disease. BioCentury mapped active pipelines across 11 leukodystrophies, highlighting where development is concentrating and how modality choices track with disease biology.

Leukodystrophies comprise more than 50 rare genetic diseases that primarily affect white matter in the brain and spinal cord, causing abnormal development or progressive loss of myelin that results in cognitive and motor impairment. ...