Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases.
A modification of the CRISPR technique has made it possible to restore vision in mouse models with retinitis pigmentosa (RP). Scientists at the Institute of Visual Neuroscience and Stem Cell Engineering of Wuhan University of Science and Technology developed a new gene-editing tool called PE(SpRY) to edit in vivo a mutation of enzyme phosphodiesterase 6B (PDE6β) and return its function.
Ophthalmic startup Eluminex Biosciences Ltd. closed a $40 million series B round to progress its pipeline of ophthalmic assets and recombinant human collagen technology. Eluminex’s pipeline includes multi-targeted antibody molecules for vision-threatening retinal diseases and an oral small molecule for rare inherited pediatric retinal dystrophies, but its lead asset, EB-301, is a biosynthetic cornea derived from recombinant human type III collagen.
Earlysight SA is developing a novel ophthalmology device that can be used to diagnose certain degenerative eye disorders long before onset of first symptoms. Earlysight and researchers at the Swiss Federal Institute of Technology Lausanne (EPFL) have reported positive results for their first clinical trial of Cellularis in the journal Ophthalmology Science.
Sparingvision SAS has obtained FDA clearance for its IND application for SPVN-06, its lead gene-independent therapy for the treatment of retinitis pigmentosa (RP). SparingVision has also submitted a clinical trial authorization (CTA) application to the French regulator (ANSM), which is currently under review.
Ocular gene therapy firm Sparingvision SA raised €75 million (US$75 million) in a series B round to fund its transition to clinical development. The company is about to move its lead program, the mutation-agnostic gene therapy SPVN-06, into a phase I trial in retinitis pigmentosa (RP). “We are in the middle of the regulatory submission process,” CEO Stéphane Boissel told BioWorld.
Researchers at the RIKEN Center for Biosystems Dynamics Research in Japan have deployed improved genetically modified human stem cell-derived retinal transplants to treat rats with retinitis pigmentosa, a major cause of hereditary human blindness.
Endogena Therapeutics Inc., of San Francisco, has raised another $20 million in a series A funding round to progress a regenerative medicine that could use stem cells to “heal” the damage caused by eye disease retinitis pigmentosa (RP).
LONDON – Intellia Therapeutics Inc. is to receive 10% of the equity in Sparingvision SAS as part of a deal giving the French ophthalmology specialist certain exclusive rights to in vivo CRISPR/Cas9 technologies in the treatment of ocular diseases.
The 2017 FDA approval of Luxturna (voretigene neparvovec-rzyl, Roche Holding AG) spurred a race to create the next gene therapy for the eye. The organ is very amenable to gene therapy given that it's a confined space with post-mitotic cells that has immune privilege and requires substantially smaller amounts of viral vector compared to systemic treatments.
