STAT3h
Gene therapies and CRISPR drugs slowed by ‘irrational pessimism,’ say scientists forging new paths
Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...
KXLY4h
Groundbreaking gene therapy research in Washington could treat children living with severe epilepsy
Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is ...
GEN - Genetic Engineering and Biotechnology News20h
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
University of California3h
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
GEN - Genetic Engineering and Biotechnology News3h
From Tools to Trials: Editing Therapy Companies Pivot to Development
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
FOX13 News on MSN4h
Breakthrough gene therapy in Seattle offers hope for children with epilepsy
The research, taking place at the Allen Institute for Brain Science, offers new hope to families fighting against Dravet Syndrome, a severe form of epilepsy that devastates childhood development.
pharmaphorum22h
Nanoscope preps filing for retinitis pigmentosa gene therapy
Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of ...
The American Journal of Managed Care9h
Dr Toon Van Gorp: Mirvetuximab Soravtansine Should Be Global Standard for FRα+ Ovarian Cancer
Toon Van Gorp, MD, PhD, a MIRASOL trial investigator, emphasizes that the final analysis reinforces the efficacy of mirvetuximab soravtansine-gynx (Elahere; AbbVie) in patients with folate receptor ...