Gene Therapy

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.

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.

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.

Investigators continue to break new ground, giving hope to patients, physicians.

Investigators observe dramatic decrease in treatment burden seen in OPTIC study.

Treatment may facilitate sight in end-stage retinitis pigmentosa.

Baseline diabetic retinopathy severity is an important predictor of its progression in patients.

Gene therapies to mitigate the effects of mutations that cause blindness are undergoing rapid development. Investigators have found that novel gene vectors reduce the risks associated with these procedures.

Study examining the role of IL-17A in patients with diabetes.

Investigators focus on biophysical method to study protein-protein interactions .

The FDA has granted Fast Track designation to MeiraGTx's AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM).