Jason Mast

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.

Johnson & Johnson said it will purchase the startup Firefly Bio for $1 billion in an effort to expand its work in the suddenly buzzy field of KRAS inhibitors.

The FDA will reconsider approving Regenxbio's experimental gene therapy for a deadly and rare childhood brain disorder it rejected just four months ago.

RIP Sangamo, onshoring with drug manufacturing, and more biotech news from The Readout