Gene Therapy

A European clinical stage biotech company announced dosing of the first patient in cohort 2 of its ongoing phase 1/2 GATEWAY clinical trial of lead candidate VTX-801 to treat Wilson’s disease.

Dosing is complete in the first cohort of the phase 1/2 clinical trial of OPGx-LCA5 for the treatment of Leber congenital amaurosis, according to a press release from Opus Genetics.

In this episode, John A. Hovanesian, MD, FACS, and Jim Mazzo discuss the latest news in eye care and chat with Wiley A. Chambers, MD, about his career in the FDA and his plans for retirement.

The FDA has approved Lenmeldy as the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy.

In this video, Diana Do, MD, discusses the impact of socioeconomic disparities on patients with diabetic eye diseases.

Durga S. Borkar, MD, MMCi, assistant professor of ophthalmology and director of clinical data science Duke University Eye Center touches on geographic atrophy therapies currently in the pipeline and how they will contribute to the future of treatment.

The FDA’s broad indication for the use of beremagene geperpavec therapy for the treatment of dystrophic epidermolysis bullosa may have large financial implications for payers, according to a study.

Ophthalmic application of beremagene geperpavec was successful in treating recurrent cicatrizing conjunctivitis in one patient with dystrophic epidermolysis bullosa, according to a study.

Mohandas Narla, DSc, may hold the title of American Society of Hematology president, but his focus for the next several months will extend far beyond this country’s borders.

Benefits of chimeric antigen receptor T-cell therapy far outweigh the risks involved, including secondary T-cell malignancies, according to Peter Marks, MD, PhD, director of the FDA’s Center for Biologic Evaluation and Research.