Passage Bio Reports the First Patient Dosing of PBKR03 in the P-I/II (GALax-C) Trial for the Treatment of Infantile Krabbe Disease
Shots:
- The 1st patient has been dosed in the P-I/II (GALax-C) trial to evaluate PBKR03 in patients with early infantile Krabbe disease. The patient enrollment is ongoing for cohort 1 globally & results from an initial safety & biomarker data are expected at the end of 2022
- The study will identify the low & high doses of PBKR03 in 2 age groups initiated with a low dose cohort of children aged 4 to 9mos. The 1EPs is to evaluate the safety & tolerability of PBKR03 & 2EPs include disease biomarkers & clinical outcome measures
- In interim results, 2 patients in the GM1 trial showed clinical improvements & safety profiles. PBKR03 received FTD, ODD & RPD designations from the US FDA & received ODD from the EU
Ref: Globe Newswire | Image: Passage Bio
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