The Phase 1/2 trial is a first-in-human, open-label, multi-national study designed to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. Trial participants will receive a ...

STRM.BIO, a biotechnology company pioneering non-viral delivery technologies for in vivo cell and gene therapy, today ...

Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, today announced Dyno-yp2, a novel adeno-associated ...

Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational ...

At ASH 2025, investigators reported early results from the first-in-human phase 1 inMMyCAR study evaluating KLN-1010, an ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...

In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory approvals worldwide for 10 therapies — all small molecules and ...

The illustration shows how high trafficking of blood stem cells in the circulation early post-natally allows lentiviral-vector mediated in vivo gene transfer. “After birth, blood stem cells need to ...

Gene-editing techniques — and particularly base editing, which allows precise editing of single nucleotides — have the potential to correct many disease-causing genetic variants. However, efforts to ...

By the end of March, the agency could approve multiple “national priority” voucher winners, as well as a gene therapy it ...

A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this ...