Fy­o­dor Urnov had giv­en a ver­sion of his speech dozens of times be­fore. Yet as he leaned in­to the mi­cro­phone to ad­dress the FDA’s top reg­u­la­tors late last year, he felt his heart rac­ing. The agency of­fi­cials were there to hear about a slow-burn­ing cri­sis: What do you do when the root cause of a dis­ease is known, the treat­ment is clear, but drug com­pa­nies won’t touch it?

It was No­vem­ber 2024, and in a Mar­riott con­fer­ence room in Bethes­da, MD, Urnov de­scribed a tongue-twist­ing and in­cred­i­bly rare con­di­tion called fa­mil­ial he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis, or HLH. Caused by one of sev­er­al ge­net­ic mu­ta­tions, it over­ac­ti­vates the im­mune sys­tem in young chil­dren. With­out a stem cell trans­plant, the chil­dren die. Even with one, the prog­no­sis is poor.

Vor Bio, found­ed by famed can­cer bi­ol­o­gist Sid­dhartha Mukher­jee, is look­ing for a way out.

Af­ter at­tempt­ing to shake up how blood can­cers are treat­ed with cell and genome en­gi­neer­ing, Vor has now de­cid­ed to end its mis­sion of cre­at­ing shield­ed trans­plants, CAR-T ther­a­pies and an­ti­body-drug con­ju­gates.

The Cam­bridge, MA-based com­pa­ny said Thurs­day that it scrapped its pair of clin­i­cal tri­als in acute myeloid leukemia and myelodys­plas­tic syn­dromes, laid off 95% of its staff, and is look­ing for an ac­quir­er or li­cen­sor of its as­sets, a merg­er or some oth­er form of busi­ness deal.

Mukher­jee har­bored big ex­pec­ta­tions for Vor's pipeline. A year ago, he told End­points News that Vor was "among the first to trans­plant hu­man bone mar­row that had been gene edit­ed." The on­col­o­gist and Pulitzer Prize-win­ning writer called it a "very ex­cit­ing time."