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Fabry recapFabry trials recap AVRO - Dosed 5 in Canada for Ph 1 beginning in early 2017 - Dosed 9 in Ph 2 from 2018-2021 - After 4 years they terminated program Freeline - Dosed 2 in 7.5e11 Ph 1 program 2019-2021 - Initial review of myocarditis in low dose led to decision to add a 3rd patient in cohort 1. - Subsequent review of data led to decision to eliminate extra patient and move to cohort 2 dose of 1.5e11 - After 3 years they have dosed 2 patients who showed subtherapeutic a-Gal with some concerning safety signals. 4D Molecular - Dosed 3 C1 1e13 in early 2021 - Added exclusion criteria for high antibody titer to AGA - Supratherapeutic aGal expression, encouraging substrate and cMRI signals. - May lower dose in next cohort tbd. Next data 1Q23 - In 1.5 years they dosed 3 with positive preliminary data. Unclear how long they will need to complete dose finding study. Sangamo - C1 0.5e13 2 dosed 2H20. Mth 15 2.7-12.4x normal a-Gal, stabile or improved LVH cMRI. One patient ERT withdraw - C2 1e13 2 dosed 1H21. 36-48 wk 4-10x aGal. 1 patient ERT withdraw a-Gal remains 10x w/moderate increase substrate. - C3 3e13 3 dosed 4Q21-2Q22. All have withdrawn ERT - C4 5e13 escalation 2 dosed no safety signals - C4 5e13 expansion 2 dosed Aug 2022 - 2 years after initial dosing they have completed dose finding study and begun expansion. No safety signals apparent. Clear signs of efficacy with supratherapeutic aGal expression, initial signs of cardiac improvement |
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Msg # | Subject | Author | Recs | Date Posted |
167532 | Re: Fabry recap | rynotheknife | 3 | 9/4/2022 7:48:53 PM |
167537 | Re: Fabry recap | Rake7613 | 17 | 9/5/2022 7:40:59 AM |
167988 | Re: Fabry recap | ZFP | 16 | 9/29/2022 7:26:22 PM |
168143 | Re: Fabry recap | ZFP | 6 | 10/12/2022 8:43:06 AM |
169352 | Re: Fabry recap | MartyCh | 14 | 12/23/2022 5:39:51 PM |