Ophthalmology

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.

Future data from other participants with LCA enrolled in the phase 1/2 study will be announced at a later date.

The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.

OCU400 utilizes Ocugen’s modifier gene therapy platform, which may allow the product to treat multiple retinal diseases.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The therapy was previously granted orphan drug designation for both RP and Stargardt disease.

Both botaretigene sparoparvovec and JNJ-81201887 were well-tolerated in treated patients, according to data from the 2022 AAO meeting.

Significant improvements were reported in full-field stimulus testing.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

NR082 has been evaluated in 3 previous investigator-initiated trials involving 186 patients.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Scientist have proposed several methods for converting stem cells into RPE, but there is still a gap in our knowledge of how cells respond to these stimuli over time.

Adverum Biotechnologies’ multicenter, double-masked LUNA trial will enroll 72 patients aged 50 years or older.

Nanoscope Therapeutics’ Phase 2 STARLIGHT open-label trial enrolled 6 subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The disease models used also helped to establish the function of NPHP5 in cilia morphogenesis.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The study’s primary end points are change in visual acuity and the occurrence of adverse events.

The therapy has demonstrated benefit in multiple models of dry AMD.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Researchers used novel functional MRI approaches to better measure the effects of gene therapy in children in current clinical trials.

While focused on the retina, the researchers showed the platform works for the identification of AAVs that target tissues in various organs.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Matthew J. Frigault, MD, discussed safety and efficacy findings on CART-ddBCMA.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.

Review top news and interview highlights from the week ending August 12, 2022.