Remember when Professor Voit said that the 25mg weekly dosage rate was in the ballpark. We now have more evidence that 25mg could be an effective therapeutic dose which is nice because of the safety profile. The plasma proteins including LTBP4 in the DMD non-ambulant cohort were increased to levels seen in the external control of healthy adults.
We are seeing a re-calibration of the target focus in disease indications from inflammation to the dual-axis of inflammation and fibrosis. It is apparent that ATL1102 is effective in targeting the TGF-ß signalling pathway that stimulates fibrosis and inhibits muscle regeneration. Its like a new avenue has been unexpectedly opened up but more importantly the patients own body is enlisted in the fight.
It is undoubtedly also of interest that the TGF-ß signalling pathway is implicated in cancer where it plays a dual role both in precancerous conditions and during tumor progression. "For the avoidance of doubt: "fibrosis" is also a key player in cancer" @Uboy
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- Ann: New data supports ATL1102s broader clinical potential
Ann: New data supports ATL1102s broader clinical potential, page-43
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