Gene Therapy

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.

Investigators are conducting retinal gene therapy trials that offer potential new treatments for inherited causes of vision loss.

An international research team has shown that optogenetic therapy has helped to partially regain visual function in a patient with retinitis pigmentosa. This is a milestone towards a gene therapy that could restore vision.

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.

In a presentation at ARVO, Friederike Kortuem, MD, MSc, explains that treatment with voretigene neparvovec-rzyl led to a short-term change in the foveal morphology in a patient with visual impairment that included nyctalopia and decreased visual acuity in early childhood.

Friederike Kortuem, MD, MSc, discusses how treatment with voretigene neparvovec-rzyl led to a short-term change in the foveal morphology in a patient with visual impairment that included nyctalopia and decreased visual acuity in early childhood.

According to Applied Genetic Technologies Corp., groups 5 and 6 had a 50% response rate among patients who met the inclusion criteria for the Skyline and Vista trials.

Investigators use an adeno-associated viral vector in a clinical trial to deliver a normal functioning copy of the RPGR gene via subretinal injection.

During an ARVO presentation, Paul Yang, MD, PhD, explained that in a clinical trial, investigators used an adeno-associated viral vector to deliver a normal functioning copy of the RPGR gene via subretinal injection.

EYS809, a non-viral gene therapy sustained drug-delivery product that delivers anti-vascular endothelial growth factor to the eye, may replace the need for repeated intravitreal anti-VEGF injections and improve vision in patients diagnosed with wet age-related macular degeneration.

Approach can unravel causes in MYOC and TBK1 glaucoma.

Studying zebrafish helps unravel mysteries of photoreceptor regeneration .

Studies are uncovering a range of potential treatment options for disorders.

Options for physicians are increasing in availability with fewer burdens for patients.

Physician offers patient counseling pearls for selected retinal diseases.

Option demonstrates stable to improved visual acuity and retinal thickness.