- Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across four dose cohorts in the nine treated patients in the dose escalation phase. - All nine patients exhibited ...

-- Unprecedented data from FORTITUDE™ dose escalation cohorts for del-brax treated participants, compared to placebo, demonstrate improvement in function, strength and PROs as well as rapid and ...

Sustained benefit demonstrated with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for nearly four years for the longest treated patient as of the data cutoff date Positive ...

Beam Therapeutics Inc. (NASDAQ: BEAM) is among the 12 Best Genomics Stocks to Invest In.

-- 30.2-month median progression-free survival with a median follow-up of 38.1 months in the Phase 1 study of anito-cel; median overall survival not reached -- -- Preliminary results from 58 patients ...

Deep, stable and durable reductions in kallikrein observed Among 32 patients who received a 50 mg dose of lonvo-z as of data cutoff: 31 (97%) were attack-free and long-term prophylaxis (LTP)-free 24 ...

PARIS--(BUSINESS WIRE)--Genethon, a pioneering gene therapy research organization created by AFM-Telethon, today presented positive results from the Phase 1/2 dose escalation part of an international ...