Jason Mast

Flush with obesity profits, Lilly is making a big push into a hot topic Eli Lilly struck a deal Tuesday to develop new forms of gene editors potentially capable of inserting entire genes into patients.

Company is one of several trying to make improved exon-skipping treatments

F For the first two years of Joshua Jacob Gonzalez's life, his parents, Javier and Jessica Gonzalez, suctioned saliva from the back of his mouth every five minutes.

Astellas renews trials of gene therapy for XLMTM, hoping for success stories like JJ's

Trial tested the first of a new wave of brain-targeted treatments Capsida Biotherapeutics said Tuesday that it still had no answers in its investigation into the death of a child in a gene therapy trial last September.

The risks of such treatments need to be weighed against what can be profound benefits, doctors say

Study of Dravet syndrome patients could raise concerns for Sarepta, Regenxbio and others

Company hopes to prove treatment is more effective, safer than Sarepta Therapeutics' Elevidys

Results deal blow to patient community and to drug developers Biogen, Denali Therapeutics

Eli Lilly said Monday that a high dose of its gene-editing therapy reduced cholesterol levels by 62% in participants in a clinical trial, an early but encouraging test of whether a one-time treatment may one day help people seeking to lower their LDL, or “bad,” cholesterol.