gene therapy
Restore Vision Doses First Retinitis Pigmentosa Patient in Phase I/II Gene Therapy Trial
The Tokyo-based biotech firm expects RV-001 to be able to alter light sensitivity for patients with limited or no vision regardless of genotype.
In Brief This Week: AstraZeneca, Arrowhead, Immix, Ensoma, Elixirgen, Ideaya Biosciences
News items for the week of Feb. 10, 2025.
Encoded Therapeutics Lays off 29 Percent of Workforce
The move will provide funds to advance ETX101, a gene therapy the company is developing for SCN1A-positive Dravet syndrome, among other programs.
Precision Medicine Supporters Worry About NIH Indirect Rate Cut's Impact on Advances, Workforce
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Researchers have been "making great inroads" in precision medicine, and a 15 percent cap on indirect costs could quash the momentum, experts worry.
Boehringer Ingelheim Strikes Deal to Use ExpressionEdits Tech in Two Gene Therapy Programs
ExpressionEdits' Genetic Syntax Engine platform is designed to enhance gene expression within gene therapies by restoring introns.