Gene Therapy

Researchers indicated that pediatric patients with Leber congenital amaurosis experienced improvements in vision.

Patients who underwent bilateral treatment experienced a higher rate of clinically relevant recovery, the authors reported.

The University of Bristol research team used animal models to compare patient responses to AAV therapy, studying male and female eye cells across different age groups (young, middle-aged and older patients)

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated inherited retinal dystrophy achieved encouraging efficacious results.

Laru-zova is a gene therapy currently being investigated for the treatment of patients with X-linked retinitis pigmentosa.

The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the genetic defects in the BBS1 gene.

The funding will support activities like formal pharmacology and toxicology testing.

ViGeneron also received approval for dose escalation in the ongoing phase 1b clinical trial.

Alon Kahana, MD, PhD, discusses the role of interleukin-6 in thyroid eye disease and the potential of tourmaline Bio's IL-6 inhibitor therapy.

Options empower patients and providers to decrease treatment burden.

The company is advancing its Phase I/II trial and exploring accelerated approval pathways in the US and Europe.

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.