FDA slaps a clin­i­cal hold on mes­sen­ger RNA pro­gram for rare ge­net­ic dis­ease

Trans­late Bio’s plans to put a sec­ond mes­sen­ger RNA drug in the clin­ic in the first half of 2019 has just ground to a halt in front of a sud­den reg­u­la­to­ry red light.

The biotech $TBIO, which went pub­lic in the class of 2018, an­nounced this morn­ing that the FDA has slapped a clin­i­cal hold on its IND for MRT5201. The IV drug is de­signed to ad­dress rare cas­es of or­nithine tran­scar­bamy­lase (OTC) de­fi­cien­cy, an X-linked ge­net­ic dis­ease.

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