Researchers expand human genome map to 2.37 million regulatory DNA elements

A research team led by Zhiping Weng, Ph.D., and Jill Moore, Ph.D."18, at UMass Chan Medical School, has nearly tripled the ...
Science Daily

Bacteria encode hidden genes outside their genome--do we?

A 'loopy' discovery in bacteria is raising fundamental questions about the makeup of our own genome -- and revealing a potential wellspring of material for new genetic therapies. A "loopy" discovery ...

Encoded Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to ETX101 for the Treatment of Dravet Syndrome

Encoded Therapeutics, Inc. ("Encoded"), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug ...
The Cardiology Advisor

AHA Scientific Statement Links Mitochondrial Genetic Variants to CVD

Management of mitochondrial-related heart disease relies on multidisciplinary care and symptom control, as targeted gene-editing therapies are limited.
Precision Medicine Online

Regenxbio Moves Toward BLA for Duchenne Gene Therapy Anticipating 2026 Pivotal Data Readout

The firm at the JP Morgan Healthcare Conference additionally expects a regulatory decision on its gene therapy RGX-121 for Hunter syndrome next month.
PharmiWeb

Encoded Therapeutics Highlights Progress in Dravet Syndrome Program and Pipeline Ahead of Company Presentation at 44th Annual J.P. Morgan Healthcare Conference

Positive interim efficacy data in 2025 underscore potential of ETX101 as a one-time, disease-modifying medicine for Dravet syndrome –– Internal GMP manufacturing capable of supporting pivotal trial ...