Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Amicus Therapeutics has two products in the market that generated more than $528 million in revenue last year.

CRISPR-based Spotlight Therapeutics has shut down, former CEO Mary Haak-Frendscho, Ph.D., confirmed with Fierce Biotech. | ...

Story Feb. 13 - Encoded Therapeutics: The gene therapy biotech is laying off 29% of staffers as it focuses resources toward its Dravet syndrome program. Most of the affected roles will be in ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...

Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) 1 and OXB, 1 today announce the start of LENTICLAIR TM 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, ...

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company focused on advancing adeno-associated ...

Rockville’s MaxCyte has made its first acquisition in its more than quarter-century of operation. It’s keeping its eyes out ...