Robert Ang, Vor Biopharma CEO

Putting a twist on tar­get­ed ther­a­py, Vor Bio­phar­ma her­alds proof of con­cept for its gene-edit­ed stem cells

Nor­mal­ly in a first-in-hu­man clin­i­cal tri­al, re­searchers are look­ing to find the right dose of their ex­per­i­men­tal drug. Vor Bio­phar­ma set out to do some­thing dif­fer­ent: Not on­ly did it want to see if its ther­a­py, a gene-edit­ed stem cell trans­plant, can en­graft safe­ly; it al­so hopes to help pa­tients bet­ter tol­er­ate an­oth­er drug known for its side ef­fects.

While ear­ly da­ta from a sin­gle pa­tient aren’t say­ing much about po­ten­tial ef­fi­ca­cy, the com­pa­ny be­lieves they do of­fer key proof of con­cept for an un­ortho­dox idea pro­posed by Co­lum­bia on­col­o­gist (and cel­e­brat­ed au­thor) Sid­dhartha Mukher­jee.

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