Sangamo Therapeutics presents updated proof-of-concept data for BIVV003 and giroctocogene fitelparvovec at ASH

SANGAMO THERAPEUTICS, INC

BIVV003 in sickle cell disease: 3 of 4 Group 1 patients maintained HbF >30% with no severe VOCs up to 30 months.
Group 2 Patient 5 achieved HbF 45% and total Hb 12.4 g/dL at week 26 using improved manufacturing.
Hemophilia A gene therapy: highest dose cohort had zero annualized bleeding rate in first year post-infusion.
Mean FVIII activity at week 156 was 25.5% of normal in highest dose cohort with no inhibitor development.
No treatment-related serious adverse events for BIVV003; one grade 3 hypotension event in hemophilia A study resolved.

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