SEVILLE, SPAIN — Ear­li­er this year, the long-held dream of per­son­al­ized gene edit­ing was made re­al with the cre­ation of a base edit­ing ther­a­py to cor­rect the unique mu­ta­tion that caused “Ba­by KJ’s” rare and oth­er­wise lethal meta­bol­ic dis­ease.

Now the sci­en­tists be­hind that break­through are ready to make more cus­tom drugs in a clin­i­cal tri­al that could be­gin next year.

Speak­ing at the Eu­ro­pean So­ci­ety for Gene and Cell Ther­a­py meet­ing in Spain on Tues­day, Ki­ran Musunuru, the Uni­ver­si­ty of Penn­syl­va­nia sci­en­tist who co-led the de­vel­op­ment of Ba­by KJ’s ther­a­py, said he re­cent­ly met with the FDA to dis­cuss a “mas­ter pro­to­col” for a Phase 1/2 tri­al that will re­cruit pa­tients with one of six urea cy­cle dis­or­ders, and could turn Ba­by KJ’s one-off ther­a­py in­to an ap­prov­able, re­peat­able pro­ce­dure.

Lawyers are field­ing in­creas­ing in­quiries from bio­phar­ma com­pa­nies on Pres­i­dent Don­ald Trump’s 100% phar­ma tar­iffs, but many are un­sure how to ad­vise their clients with gap­ing holes in the ad­min­is­tra­tion's an­nounce­ments.

“There's a lot of chat­ter be­hind the scenes and at­tempts to un­der­stand and get clar­i­ty on how the next it­er­a­tion spells out,” Lynn Mehler, phar­ma reg­u­la­to­ry lawyer at Lon­don-head­quar­tered firm Hogan Lovells, told End­points News.

Trump on Sept. 25 an­nounced via so­cial me­dia that bio­phar­ma com­pa­nies not start­ing con­struc­tion projects in the US by Oct.1 would face 100% tar­iffs on brand­ed drugs. But on Oct. 1, a White House of­fi­cial con­firmed to End­points these levies would be de­layed, as they are still be­ing pre­pared.

Vinay Prasad, the FDA's bi­o­log­ics chief, told staffers on Mon­day about the lat­est changes to lead­er­ship in his cen­ter, af­ter he abrupt­ly left and then re­turned to the agency in Au­gust.

Kather­ine Szara­ma, who joined ARPA-H in Jan­u­ary, will take over as act­ing deputy di­rec­tor of CBER, Prasad wrote to staff in an email ob­tained by End­points News. "She will fo­cus on pol­i­cy and gov­ern­ment re­la­tions, but al­so par­tic­i­pate more broad­ly at the cen­ter," he wrote.

Szara­ma will take over for Brit­tany Gold­berg, who will move over to chief med­ical of­fi­cer at CBER.

"Brit­tany is hard-work­ing and bril­liant," Prasad wrote. "This shift will al­low her to hand off the ad­min­is­tra­tive du­ties of CBER, and fo­cus more on the med­ical themes and is­sues, where we ben­e­fit from her wis­dom."