Gene Therapy

Mariya Moosajee, MBBS, BSc, PhD, FRCOphth, discusses how Gene Vision is improving patients' lives on a global scale

Researchers from the University of Bristol recorded increased instances of inflammation in particular demographic groups

OCU400 is the first gene therapy from the biotechnology company to move forward into Phase 3 with a broad retinitis pigmentosa (RP) indication

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 lead gene therapy candidate, AXV-101, is expected to enter clinical development in mid-2025

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 company, which has European headquarters in Basel, Switzerland, plans to use the funds for a non-interventional, observational study and a first-in-human clinical trial

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.

ViGeneron, based in Munich, also received approval for dose escalation in the ongoing phase 1b clinical trial

The RESTORE Study is the follow-up study of the RESCUE and REVERSE studies of lenadogene nolparvovec to treat vision loss from LHON

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.

Our top 10 print stories highlight the core content concepts we explored this year

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

Ocular clues to a neurological conditions help clinicians understand neuromyelitis optica spectrum disorder (NMOSD)

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.

Phenocell and Amarna Therapeutics were awarded the grant by Eureka’s Eurostars initiative, co-funded by the European Union

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.

In an interview with Ophthalmology Times, Alon Kahana, MD, PhD, discusses interleukin-6 and its role in autoimmune diseases, particularly thyroid eye disease, noting that a promising alternative under clinical trial is pacibekitumab, an IL-6 ligand-blocking antibody.

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.