Gene Therapy

In clinical ophthalmology, we are used to fixing problems on a physical or mechanical level.

It has been decades since the concept of gene therapy emerged in the ophthalmology field.

Most ophthalmologists are familiar and comfortable with the application of devices and drugs in their daily practice.

Avista Therapeutics and Roche will partner to develop adeno-associated virus gene therapy vectors for ocular diseases, according to a press release.

The first patient has been dosed in a phase 2 clinical trial investigating MCO-010 ambient light activatable optogenetic monotherapy for Stargardt disease, according to a press release from Nanoscope Therapeutics.

A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who received the investigational gene therapy for moderate to severe hemophilia B, study results showed.

A phase 3 trial of ofranergene obadenovec in platinum-resistant ovarian cancer failed to meet its primary endpoints of statistically significant improvement in PFS and OS, according to topline data released by the agent’s manufacturer.

Gene therapy company uniQure has announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130 for treatment of Huntington’s disease.

Botaretigene sparoparvovec, an investigational gene therapy, was safe and improved vision in subjects with X-linked retinitis pigmentosa with disease-causing variants in the RPGR gene, according to a press release from MeiraGTx.

Biotechnology company bluebird bio announced that an FDA advisory committee has unanimously endorsed its gene therapy treatment for early active cerebral adrenoleukodystrophy.