Clin­i­cal hold for Pep­Gen's my­oton­ic dy­s­tro­phy pro­gram halts march to the clin­ic

The FDA im­posed a clin­i­cal hold on Pep­Gen’s IND for a drug for my­oton­ic dy­s­tro­phy type 1 (DM1), slow­ing down the com­pa­ny’s plans to bring a sec­ond neu­ro­mus­cu­lar pro­gram in­to the clin­ic.

Pep­Gen did not spec­i­fy what’s caus­ing con­cern, say­ing on­ly that the FDA will pro­vide an of­fi­cial let­ter with­in 30 days stat­ing the rea­sons be­hind the hold.

Spun out of Ox­ford Uni­ver­si­ty and backed by RA Cap­i­tal, Pep­Gen spe­cial­izes in oligonu­cleotides — short strands of syn­thet­ic RNA or DNA mol­e­cules — that it de­liv­ers with spe­cial cell-pen­e­trat­ing pep­tides. By con­ju­gat­ing the pep­tides with oligonu­cleotides, the biotech promis­es to im­prove the up­take and ac­tiv­i­ty of the re­sult­ing ther­a­py.

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