An eye-drop version of Krystal Biotech’s skin gene therapy helped restore vision of boy with rare disease

A re­for­mu­lat­ed ver­sion of Krys­tal Biotech’s gene ther­a­py for “but­ter­fly skin” helped re­store the vi­sion of a boy with a rare dis­ease, which can cause scar­ring of the eye tis­sue in some pa­tients.

The 13-year-old boy — An­to­nio, ac­cord­ing to the AP — has a rare ge­net­ic dis­ease called dy­s­troph­ic epi­der­mol­y­sis bul­losa, or DEB, that dis­rupts the pro­duc­tion of a type of col­la­gen. It leads to skin that’s ex­treme­ly frag­ile and that can blis­ter or tear, like the wings of a but­ter­fly. Around a quar­ter of these chil­dren al­so ex­pe­ri­ence scar­ring of the eye, ac­cord­ing to Krys­tal, which can po­ten­tial­ly re­sult in blind­ness.

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