Gene Therapy

FORT LAUDERDALE, Fla. — Best practices for safe subretinal delivery of gene therapy were presented at the Retina World Congress.

DENVER — Surgical delivery of the genetic therapy AGTC-401 resulted in overall improvement on a test of light discomfort in 40% of patients with achromatopsia, according to a study presented at the ARVO meeting.

The FDA granted fast track designation to ofranergene obadenovec in combination with paclitaxel for treatment of platinum-resistant ovarian cancer.

The FDA and European Commission have both granted orphan drug designation to AviadoBio for its new gene therapy to treat frontotemporal dementia, according to a company press release.

SEATTLE — Clinicians should consider disease-modifying therapeutics when treating patients with rare pediatric disorders, according to an expert at the 2022 American Academy of Neurology annual meeting.

Gene therapy appeared safe and effective for children with Wiskott-Aldrich syndrome without a suitable donor for hematopoietic stem cell transplantation, according to a study published in Nature Medicine.

Valoctocogene roxaparvovec induced endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis in patients with severe hemophilia A, according to study results.

Gene therapy has been around for decades now, but it has had a history of challenges with only a few breakthroughs.

Genomic medicine encompasses a wide variety of interventional strategies.

In recent decades, ophthalmology has been a leader in exploring cutting-edge technologies for combating blinding diseases, and innovative gene-based strategies have shown promising results.