Tout­ing 100% OS rate in piv­otal rare dis­ease tri­al, Rock­et Phar­ma ready to head to FDA

In drug de­vel­op­ment, you don’t throw around the word “cure” light­ly. But Rock­et Phar­ma­ceu­ti­cals’ lat­est cut of gene ther­a­py da­ta has at least one an­a­lyst won­der­ing about the cu­ra­tive po­ten­tial.

Re­port­ing topline re­sults from a Phase II piv­otal tri­al in­volv­ing pa­tients with se­vere leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-1), Rock­et said among nine par­tic­i­pants, the over­all sur­vival at one year is 100%.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER