Bridge­Bio dou­bles down on rare dis­ease with CoA launch; Be­lea­guered scPhar­ma­ceu­ti­cal­s' shares wilt again on FDA's re­jec­tion let­ter

→ In its sec­ond rare dis­ease start­up launch in a mat­ter of days, SF’s Bridge­Bio is un­veil­ing a new ven­ture Wednes­day that’s set to de­vel­op up a trunk of as­sets it li­censed from St. Jude Chil­dren’s Re­search Hos­pi­tal. The com­pa­ny, called CoA Ther­a­peu­tics, is de­vel­op­ing small mol­e­cules to in­crease Coen­zyme-A lev­els in rare ge­net­ic dis­or­ders that in­volve a de­fi­cien­cy of the en­zyme. The com­pa­ny’s lead com­pound will fo­cus on pan­tothen­ate ki­nase-as­so­ci­at­ed neu­rode­gen­er­a­tion (PKAN), an au­to­so­mal re­ces­sive ge­net­ic dis­or­der re­sult­ing from mu­ta­tions in the pan­tothen­ate ki­nase 2 (PANK2) gene es­ti­mat­ed to af­fect three in every mil­lion peo­ple.

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