Gene Therapy

In a study, investigators demonstrated the safety and therapeutic potential of clinically compliant iPSC-derived photoreceptor precursors as a cell replacement source for future clinical trials.

Results from Aurion Biotech's IOTA trial reveals that a corneal endothelial cell therapy showed improvements in visual acuity and central corneal thickness, according to a team of Japanese investigators.

Richard B. Rosen, MD, DSc(Hon), reports on the capabilities of using clinical OCT for imaging and measuring macular surface macrophage cells during the 2021 ASRS annual meeting.

According to 4D Molecular Therapeutics, it has received FDA Clearance of an IND Application for 4D-150, a dual-transgene intravitreal gene therapy for patients with wet AMD.

A team of investigators have found that a molecular mechanism drives the progression of uveal melanoma, an often lethal eye cancer in adults.

AbbVie and REGENXBIO announced a strategic partnership to develop and commercialize RGX-314, an investigational gene therapy for wet age-related macular degeneration, diabetic retinopathy and other chronic retinal diseases.

José-Alain Sahel, MD, explains how optogenetics involves artificial photoreceptors from specific retinal cells to restore vision.

The gene-based AAVCOVID vaccine employs a unique adeno-associated viral vector technology that was shown in non-clinical, nonhuman primate studies to enable protection from SARS-CoV-2 challenge and to induce sustained antibody and cellular immune responses from a single dose for up to a year.

Development of new animal model enhances study of potential mutations.

Study examines immunomodulatory therapy as a potential long-term treatment option.

In PIONEER study, investigators outline their successes.

GT005 designed to induce local production of recombinant complement factor I.

CAM’s regenerative healing means long-term relief and fewer visits to clinic.

Cotargeted therapy could help combat inherited retinal diseases.

Clinical trials show subretinal agents are safe, well tolerated by patients.

As soon as 2 weeks after treatment, changes occur to foveal layer.

Investigators uncovered evidence that CaMKII could prove to be a desirable therapeutic target for vision preservation in conditions that damage the axons and somas of retinal ganglion cells.

Nanoscope Therapeutics Inc. has dosed its first patient in a late-stage Phase 2b trial of a gene therapy that delivers multi-characteristic opsin to retinal cells.

The primary barriers to COVID-19 vaccine development are manufacturing constraints.

Usher syndrome 1G protein SANS regulates the splicing of genes, particularly those linked to Usher syndrome, which can lead to early vision loss form RP.

According to Unity Biotechnology, its trial for the small molecule inhibitor of Bcl-xL found improvement in visual acuity and central subfield thickness observed in diabetic macular edema and wet age-related macular degeneration patients treated with UBX1325.

Trial shows treatment has acceptable safety profile, may improve visual function.

Investigators find approach improved vision and visual fields in some patients.

Details enhance knowledge of biological pathways that contribute to disease pathogenesis.

RGX-314 eyed by investigators as a therapeutic option for exudative AMD.

Nanoscope Therapeutics Inc. expects to advance the therapy by launching a late-stage Phase 2b trial this summer with gene therapy that delivers multi-characteristic opsin to retinal cells.

According to the company, preclinical study results from a 42-day proof-of-concept in vivo study demonstrated a single, intralacrimal gland injection of an adeno-associated virus containing the NGF gene produced statistically significant increase in NGF in tear film, as compared to placebo, as early as Day 7.

ADVM-022 offers sharp decrease in treatment burden for patients with nAMD.

While Biogen’s XIRIUS study did not meet its primary endpoint of demonstrating a statistically significant improvement in treated eyes, positive trends were observed across several clinically relevant prespecified secondary endpoints.

Mark Pennesi, MD, PhD, reports during ARVO 2021 that preliminary results have shown that AGTC-401 and AGTC-402 seem safe and well tolerated in patients with ACHM.