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 ...
A research team led by Zhiping Weng, Ph.D., and Jill Moore, Ph.D."18, at UMass Chan Medical School, has nearly tripled the ...
A newly discovered code within DNA -- coined 'spatial grammar' -- holds a key to understanding how gene activity is encoded in the human genome. This breakthrough finding revealed a long-postulated ...
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 ...
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 ...
The firm at the JP Morgan Healthcare Conference additionally expects a regulatory decision on its gene therapy RGX-121 for Hunter syndrome next month.
Management of mitochondrial-related heart disease relies on multidisciplinary care and symptom control, as targeted gene-editing therapies are limited.
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