Gene Therapy

XLRP often leads to blindness by middle age with no treatment options currently available

Investigators say the discovery highlights the importance of genetic consultation

Simply slowing disease progression is no longer enough

The IND approval will allow the company to initiate a Phase I/IIa clinical trial for its gene therapy treatment targeting wet Age-related Macular Degeneration (AMD) including Polypoidal Choroidal Vasculopathy (PCV).

Ocugen is developing OCU410 as a 1-time gene therapy for the treatment of geographic atrophy. It utilizes an AAV delivery platform for the retinal delivery of the ROR Related Orphan Receptor A gene.

Prevent Blindness provides free resources on Stargardt disease, including a webpage, fact sheets, expert interview video and social media graphics, to educate the public and professionals on the inherited retinal disease.

Researchers note the G90D mutation in rhodopsin is known to produce background electrical noise to desensitize rods, but the nature of the ‘noise’ and its precise molecular source have not been resolved for almost 30 years.

Simply slowing disease progression is no longer enough.

Promising innovations are not one-size-fits-all

Despite recognition of lenticular glutathione’s importance in lens formation for decades, why it declines with age remains a mystery.

The data will be presented in oral session at the 2024 American Society of Gene & Cell Therapy meeting May 7-11 in Baltimore, Maryland.

Program cochair Arjan Hura, MD, previews this year’s extended schedule with discussions on retina gene therapies, corneal endothelial diseases, and oculoplastics, along with hands-on experiences.

CRISPR manipulation presents opportunities and challenges.

Shift could lead to changes in the current management of retinal diseases

The tissue serves as a testing ground for outpatient ocular therapies

The Phase 3 study will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic.

Rich Small, CEO of Neurotech Pharmaceuticals, provides an update on the months ahead for NT-501 encapsulated cell therapy

The opportunities and challenges presented by CRISPR manipulation

In a recent study led by Steven Pittler, PhD, and his team at the University of Alabama Birmingham (UAB), the role of modifier genes in retinitis pigmentosa type 59 (RP59) was meticulously examined.

OCU410 is a modifier gene therapy candidate being developed for geographic atrophy, an advanced stage of dry age related macular degeneration.

In the study, published in Nature Biotechnology, researchers used their new system to correct disease-causing mutations in the eyes of two mouse models of genetic blindness, partially restoring their vision.

According to the company, its proprietary non-viral gene therapy platform with minimally invasive delivery technology is providing long lasting gene expression and favorable distribution in the retina.

ECP105 has been developed for the recovery of post-surgical treatment of glaucoma.

The trial of esonadogene mvoparvovec (Opvika) will take place in the US for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).