Gene Therapy

In the LIGHTHOUSE study, Atsena Therapeutics is evaluating ATSN-201 gene therapy for X-linked retinoschisis, leveraging AAV.SPR capsid for central retina transduction without foveal detachment risks.

USC is leading a research team aiming to better understand retinitis pigmentosa and inform future treatments.

VisiRose, a company formed in collaboration with Provectus Biopharmaceuticals and the University of Miami Miller School of Medicine’s Bascom Palmer Eye Institute, is advancing Rose Bengal Photodynamic Antimicrobial Therapy (RB PDAT), a non-invasive treatment for infectious keratitis, leveraging light-based technology and addressing antimicrobial resistance to improve outcomes for patients with severe eye infections globally.

Nona Biosciences has partnered with Kodiak Sciences to develop multi-target antibodies for ophthalmic diseases. Utilizing Nona's Harbour Mice platform, the collaboration aims to accelerate antibody discovery, focusing on innovative therapies for retinal and other eye conditions.

Preclinical research highlights mRNA-based therapy's potential to inhibit scar tissue and abnormal blood vessel growth in proliferative vitreoretinopathy, addressing a critical unmet need in treatment.

ABI-110 has the potential to offer a durable and effective solution by addressing the root causes of wet AMD at the genetic level.

Today’s technology proves faster, better, cheaper.

Atsena Therapeutics and Nippon Shinyaku have formed an exclusive licensing agreement for ATSN-101, a gene therapy for Leber congenital amaurosis (LCA1). Nippon Shinyaku will commercialize ATSN-101 in the U.S. and Japan, while Atsena retains global rights outside these territories.

The drug is the world’s first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD).

The company will seek a strategic partner to continue development of APX3330, an oral small-molecule inhibitor of Ref-1 for the treatment of non-proliferative diabetic retinopathy.

According to the company, its data replicate and expand upon the potential of ER-100 to improve retinal ganglion cell function and restore visual function in a nonhuman primate model of nonarteritic anterior ischemic optic neuropathy.

The retina is a key proving ground for long-held theories of genetics.

In data presented at the 128th annual meeting of the American Academy of Ophthalmology in Chicago, the two-year study finds patients with advanced disease can recover some vision.

Researchers at the Karolinska Institute continue to investigate whether gene therapy can improve vision in people with Bothnia dystrophy, a form of hereditary blindness, prevalent in the region Västerbotten in Sweden.

According to research conducted by investigators at the Perelman School of Medicine at the University of Pennsylvania School of Medicine, patients with a rare inherited disease affecting their sight experienced quick vision improvements that sustained for the full year-long study.

Research by investigators at Trinity College in Dublin shows how the gene therapy conferred significant benefit in animal models, and in human cells derived from people with glaucoma.

The one-time intravitreally delivered gene therapy is for the treatment of retinitis pigmentosa (RP).

The product candidate is a modifier gene therapy for broad retinitis pigmentosa indication.

According to the company, the priority review voucher would be issued upon approval of ATSN-201, a gene therapy product candidate.

The program is for candidate OCU400, a modifier gene therapy product candidate.

Researchers note work may lead to targets for conventional outflow pathway therapies.

According to the company, OCU410 utilizes an adeno-associated virus platform for the retinal delivery of the RORA gene.

AAV204 is a novel adeno-associated virus (AAV) capsid from the AIM capsid library licensed by Abeona from the University of North Carolina at Chapel Hill.

The funding will go toward the continued clinical development of the company’s lead asset, AGTC-501 (laruparetigene zovaparvovec)

Phase 1 multicenter study demonstrates early signs of efficacy.

OCU410ST is a modifier gene therapy candidate being developed for Stargardt disease, which affects approximately 100,000 people in the United States and Europe combined.

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.