Gene Therapy

Preclinical biotech company Eikonoklastes Therapeutics and gene therapy manufacturer Forge Biologics announced a partnership that will advance Eikonoklastes’ ET-101 gene therapy into clinical trials for patients with ALS.

NEW YORK — In this video, David Eichenbaum, MD, director of research at Retina Vitreous Associates of Florida, discussed the current pipeline of treatments for geographic atrophy at OSN New York Retina.

The FDA approved etranacogene dezaparvovec-drlb, a single-dose gene therapy for treatment of adults with hemophilia B.

Lysogene has announced topline results from its phase 2/3 study evaluating LYS-SAF302, an investigational gene therapy for the treatment of mucopolysaccharidosis type IIIA, or Sanfilippo syndrome.

NEW YORK — In this Healio Video Perspective from OSN New York Retina, Lejla Vajzovic, MD, FASRS, discusses the growing role of intravitreal and subretinal gene therapy in treating retinal diseases.

CHICAGO – Jeffrey Chamberlain, PhD, professor of neurology at the University of Washington, discussed types of gene therapy as a possible treatment for muscular dystrophy at ANA 2022.

CHICAGO — In this Healio Video Perspective from the AAO meeting, Michael Nathan Cohen, MD, discusses phase 1 data investigating JNJ-81201887 gene therapy from Janssen for the treatment of geographic atrophy.

CHICAGO — In this Healio Video Perspective from Retina Subspecialty Day at the AAO meeting, Michel Michaelides, MD, discusses study results from a gene therapy trial for RPGR-associated X-linked retinitis pigmentosa.

CHICAGO — In this Healio Video Perspective from Eyecelerator@AAO, Opus Genetics CEO Ben Yerxa discusses an upcoming investigational new drug application for OPGx-001.

The FDA granted accelerated approval to bluebird bio Inc for elivaldogene autotemcel, its gene therapy that aims to slow progression of neurologic dysfunction in boys aged 4 to 17 with early, active cerebral adrenoleukodystrophy, or CALD.