Gene Therapy

In this Healio Video Perspective, Sue Washer, president and CEO of AGTC, discusses the company’s trial pipeline, including a look at recently released data for an achromatopsia candidate.

In a video interview, Rishi P. Singh, MD, staff surgeon at Cleveland Clinic Cole Eye Institute, associate professor of ophthalmology at Lerner College of Medicine and Healio/OSN Board Member, discusses exploring gene therapy in intermediate and advanced stages of dry AMD.

AGTC plans to advance its achromatopsia B3 candidate to the next stage of development after 12-month phase 1/2 trial data showed positive biologic activity, according to a company press release.

World Sickle Cell Day is observed each year on June 19.

Three patients with sickle cell disease who received the investigational gene therapy ARU-1801 achieved and maintained normal hemoglobin levels, according to early results of a phase 1/phase 2 trial.

WAILEA, Hawaii — Retinal pigmentary changes were seen in 67% of patients in the phase 1/2 safety study of RGX-314, a gene therapy delivered subretinally for the treatment of AMD.

Michel Sadelain, MD, PhD, will receive this year’s Outstanding Achievement Award from the American Society of Gene and Cell Therapy for his research on chimeric antigen receptors.

A suspected unexpected serious adverse reaction has occurred in the phase 2 INFINITY trial investigating ADVM-022 gene therapy for the treatment of diabetic macular edema, according to a press release from Adverum Biotechnologies.

FDA approval of the first gene therapy in December 2017 marked the beginning of a new chapter in medical practice.

The novel gene therapy BAX 335 did not induce sustained factor IX expression among patients with hemophilia B, according to an interim analysis of a phase 1/phase 2 dose-escalation study published in Blood.