Gene Therapy

Explore the challenges and insights in long-term follow-up of paediatric gene therapy, focusing on data integrity, patient retention, and ethical considerations.

The EXTEND trial provided 5-year follow-up for 10 patients with advanced retinitis pigmentosa, who had previously received MCO-010 during a phase 1/2a trial

SPVN20 is a gene-agnostic, intravitreal AAV-based gene therapy that targets dormant cones to restore vision, according to SparingVision

A mutation-agnostic gene therapy approach offers new possibilities for patients, especially those with inherited retinal diseases

Presentation topics will include therapies for age-related macular degeneration, X-linked retinitis pigmentosa and other retinal pathologies

AMD treatment may become unrecognisable as gene therapies evolve

In a press release, the National Institute for Health and Care Excellence announced the first-ever approval for a mitochondrial disease treatment

The Global RDH12 Alliance unites inherited retinal disease advocacy groups including UK-based Eyes on the Future and the US-based RDH12 Fund for Sight

The platform, developed by University College London and Heidelberg Engineering, demonstrated accuracy and precision in early tests

Insights from a genome-wide association study identify novel genetic loci

The company shared results from the AAVB-081 clinical programme and AAVB-039 preclinical programme at ARVO

Opus Genetics reveals promising 1-year results from OPGx-LCA5 gene therapy, showing sustained vision improvements in adults with LCA5 retinal degeneration.

The company's therapeutic candidates include a bispecific antibody to target vascular retinal diseases

The partnership will use ocular imaging to build an AI foundation model and identify new genetic targets for degenerative diseases

SpliceBio continues to actively enroll patients in the ASTRA study and accompanying POLARIS trial

A large-scale study indicates women are more likely to develop the inherited corneal condition

The modifier gene therapies from Ocugen target geographic atrophy and Stargardt disease

The authors said their results provide strong support that cultivated autologous limbal epithelial cell transplantation is safe and feasible

Researchers indicated that paediatric patients with Leber congenital amaurosis experienced improvements in vision

The company shared 5-year results for lenadogene nolparvovec (Lumevoq), administered unilaterally and bilaterally to patients with Leber Hereditary Optic Neuropathy

Mariya Moosajee, MBBS, BSc, PhD, FRCOphth, discusses how Gene Vision is improving patients' lives on a global scale

Researchers from the University of Bristol recorded increased instances of inflammation in particular demographic groups

OCU400 is the first gene therapy from the biotechnology company to move forward into Phase 3 with a broad retinitis pigmentosa (RP) indication

The lead gene therapy candidate, AXV-101, is expected to enter clinical development in mid-2025