milasen

Milasen is a custom-designed antisense oligonucleotide (ASO) drug created specifically for a single patient, Mila Makovec, who suffered from an ultra-rare form of Batten disease (CLN7-related neuronal ceroid lipofuscinosis). The drug was developed in record time—about 10 months—from genetic diagnosis to first injection. Milasen works by targeting and masking a unique splicing defect in the MFSD8 (CLN7) gene caused by a retrotransposon insertion, thereby restoring normal RNA splicing and partially rescuing protein function. The approach was inspired by the mechanism of Spinraza, another ASO that modulates RNA splicing. Milasen is not suitable for other patients due to its highly individualized sequence but has set precedent for n-of-1 precision medicines in rare genetic diseases[1][2][5][6].

Brand names

milasen

Other names

milasen