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.
HONG KONG – Yongin, South Korea-based Rznomics Inc. is on track to develop adenoviruses for gene therapies, after signing a clinical and commercial agreement with Cologne, Germany based-Cevec Pharmaceuticals GmbH. The partnership will use the German company’s Cap technology to manufacture the adenoviruses, while the therapies, targeting various cancer indications, will be developed using Rznomics’ trans-splicing ribozyme technology.
LONDON – Sparingvision SAS has raised €44.5 million (US$52.5 million) to advance SPVNo6, a gene therapy designed to be effective for all retinitis pigmentosa patients regardless of the underlying mutation, with a phase I dose-ranging study now due to start in 2021.
HONG KONG – Yongin, South Korea-based Rznomics Inc. is on track to develop adenoviruses for gene therapies, after signing a clinical and commercial agreement with Cologne, Germany based-Cevec Pharmaceuticals GmbH. The partnership will use the German company’s Cap technology to manufacture the adenoviruses, while the therapies, targeting various cancer indications, will be developed using Rznomics’ trans-splicing ribozyme technology.
Keen to address a rare vision-limiting disorder endured by nearly 1.9 million people globally, ophthalmology specialist Santen Pharmaceutical Co. Ltd. has negotiated a $252 million ex-U.S. licensing deal for Jcyte Inc.'s Jcell, a human retinal progenitor cell therapy initially aimed at treating retinitis pigmentosa.
