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Base-Editing Therapy tBE Shows Durable Remission Across Ethnic Groups

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Base-Editing Therapy tBE Shows Durable Remission Across Ethnic Groups

Shanghai – September 12, 2026 -- CorrectSequence Therapeutics (Correctseq) reported that its transformer Base Editor (tBE) platform achieved sustained transfusion independence or elimination of vaso-occlusive crises (VOCs) in four new patients from Nigeria, Laos, Malaysia and Pakistan, extending clinical validation beyond the Chinese cohort where five patients previously achieved full transfusion independence.

Study published in Cell Stem Cell confirms cross-genetic efficacy of CS-101/CS-206

The peer-reviewed paper, published online September 7, 2026, details results from one sickle cell disease (SCD) patient and three transfusion-dependent β-thalassemia (TDT) patients treated with CS-101/CS-206. All four achieved rapid hematopoietic reconstitution, sustained high-level pancellular fetal hemoglobin (HbF) expression, and no detectable off-target edits or product-related adverse events.

REGENXBIO to Present at Baird, Morgan Stanley Healthcare Conferences

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REGENXBIO to Present at Baird, Morgan Stanley Healthcare Conferences

Rockville, Maryland – September 11, 2026 -- REGENXBIO Inc. (Nasdaq: RGNX) will hold fireside chats at two major investor healthcare conferences in New York this month.

REGENXBIO confirms back-to-back fireside chats on September 15 and 16

The gene therapy company will participate in the Baird 2026 Global Healthcare Conference on Tuesday, September 15 at 8:30 a.m. ET, followed by the Morgan Stanley 24th Annual Global Healthcare Conference on Wednesday, September 16 at 8:30 a.m. ET. Both sessions will take place in New York, NY.