BioSpace

FDA’s New Cell and Gene Therapy Guidances Finally Go a ‘Click Further’ to ‘Game Changing’

Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways ...

Taysha Gene Therapies: Shares Are Soaring On FDA Alignment, But Caution Is Advised

Taysha Gene Therapies stock surges after FDA alignment on a pivotal Rett Syndrome study. Read the latest analysis on TSHA ...

Stabilization of neuropathy scores seen after gene editing therapy for rare nerve disease

University College London's National Amyloidosis Center leads a multinational team reporting that a single infusion of an in vivo gene-editing therapy (nexiguran ziclumeran) produced rapid, deep, and ...
MedPage Today on MSN

Gene-Editing Therapy Promising for Rare Hereditary Disease

Using CRISPR-Cas9 gene editing technology, nex-z was designed to reduce serum transthyretin levels by selectively ...
Medscape

Mutation-Agnostic Gene Therapy May Restore Vision in RP

The MCO-010 vector uses surviving cells in the retina to produce a photosensitive protein that may help patients regain some ...
Managed Healthcare Executive

Outcomes Model Is Starting With Gene Therapy for Sickle Cell Disease

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

Preliminary but promising gene therapy shows 75% reduction in Huntington's disease progression

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers.

Study: Gene predicts breast cancer risk when abnormal cells are found

Researchers said a genetic risk score can help predict invasive breast cancer risks for women after abnormal cells have been found in their breast tissue.
Science-Based Medicine

FDA approves novel gene therapy delivery platform for a rare retinal disorder

Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative ...