2019年7月1日 · CD59 is a naturally occurring inhibitor of the assembly of MAC. CD59 may thus be therapeutically efficacious against the pathophysiology of dry and wet AMD. The first gene therapy clinical trial for geographic atrophy - the advanced form of dry AMD has recently completed recruitment.

Accounting for nearly 90% of AMD, dry AMD still lacks effective treatment. Numerous genetic and molecular researches have confirmed the significant role of the complement system in the pathogenesis of AMD, leading to a deeper exploration of …

Evidently, an overactive complement system plays a pivotal role in dry AMD development, and subsequently, GA disease progression. The accumulation of MAC on cell surfaces can lead to damage and atrophy, causing disease. CD59, a cell surface protein, blocks the formation of MAC, which is a pivotal step in complement-mediated cell lysis.

2022年8月1日 · Based on preclinical data, it was concluded that a subretinal injection of adenovirus CD59 (AAV-CD59) could offset laser-induced CNV by 60% in murine models 50; a Phase I proof-of-concept study (NCT03585556) is in progress.

In this review, we provide an understanding of the role of the complement system in dry AMD and discuss the emerging therapies in early phase clinical trials. The tentative hope is that these drugs may offer the potential to intervene at earlier stages in dry AMD pathogenesis, thereby preventing progression to late disease.

No therapy is available to slow the progression of early-stage, or dry, AMD, which accounts for approximately 90% of AMD cases. We envision that because dry AMD progresses slowly over many years, its treatment will potentially require regular injections of anticomplement molecules in patients with relatively good visual acuity.

2023年3月1日 · A phase 1, open-label, multicenter, dose-escalating, safety and tolerability study investigated intravitreal delivery of AAVCAGsCD59, which increases retinal cellular expression of a soluble, recombinant CD59 molecule. 11 CD59 occurs naturally in the body and inhibits the MAC, preventing complement-mediated cell lysis. The phase 1 trial ...

2011年4月28日 · Here we demonstrate that subretinal delivery of an adenovirus vector expressing a transgene for a soluble non-membrane binding form of human CD59 can attenuate the formation of laser-induced choroidal neovascularization and murine MAC formation in mice even when the region of vector delivery is distal to the site of laser induced CNV.

2023年6月1日 · Hemera Biosciences has developed AAVCAGsCD59/HMR59 that underwent clinical trial (NCT03144999) for dry age-related macular degeneration (AMD). The drug induces CD59 formation to prevent MAC formation.

2014年1月1日 · Divided into two forms, AMD is classified as “dry” and “wet”. Dry AMD results in atrophy of the retinal pigment epithelium (RPE), and eventually leads to photoreceptor loss [1, 2]. Patients with this form of AMD exhibit drusen, deposits found on the basal surface of the RPE . Wet AMD is so called due to subretinal fluid leakage, and is ...