Nanoscope Therapeutics licenses optogenetic technology

Technology offers hope for restoring vision for genetic visual impairments

The US biotech company Nanoscope Therapeutics has acquired a license for CatCh technology from Max Planck Innovation, the technology transfer organization of the Max Planck Society. Based on the research results of the Max Planck Institute for Biophysics and the pioneering work of Ernst Bamberg in the field of optogenetics, this patented technology improves the light sensitivity of an opsin protein (MCO-010). This is a complex consisting of three proteins including CatCh. It was developed by Nanoscope as a gene therapy to restore vision in patients with genetic visual impairments. The MCO platform is the first optogenetic approach that combines multiple light-sensitive components. The resulting fusion protein is sensitive to high-speed ambient light at all visible wavelengths. Nanoscope has already successfully completed several clinical studies of MCO-010 for the two most common inherited degenerative retinal diseases, retinitis pigmentosa and Stargardt’s disease. The company now plans to obtain approval for the therapy and expand it to additional therapeutic indications.

Genetic eye diseases such as retinitis pigmentosa, Stargardt’s disease and age-related macular degeneration represent a major medical challenge worldwide. They often lead to progressive loss of vision and even blindness and have a significant impact on the quality of life of those affected.

In many cases, these diseases cannot be adequately treated. With its ability to partially restore vision through gene therapy, Nanoscope’s MCO technology platform could represent a breakthrough in the treatment of these serious eye diseases.

Therapeutic application and clinical testing

The now licensed CatCh technology was integrated by Nanoscope Therapeutics as one of three subunits of the MCO-010 fusion protein. Each of these subunits has complementary light-activating properties. In this way, the technique becomes a treatment approach that could restore vision in everyday situations. Nanoscope’s novel MCO-010 therapeutic approach has already been successfully tested in several clinical studies for retinitis pigmentosa and Stargardt’s disease. In addition, the MCO platform has already been tested and proven effective in non-human primates with geographic atrophy secondary to advanced age-related macular degeneration.

The further development and approval path for MCO-010 is currently being intensively examined. Nanoscope Therapeutics is seeking approval and is in discussions with the US Food and Drug Administration to explore possible accelerated routes to market. These efforts could help make the therapy more quickly available to patients who urgently need new treatment options.

From basic research to application

The CatCh (calcium-transporting channelrhodopsin) technology was developed by Ernst Bamberg, one of the pioneers in the field of optogenetics, and his colleagues at the Max Planck Institute for Biophysics. CatCh, a mutant of channelrhodopsin-2 with improved properties, offers key advantages such as higher speed and higher sensitivity to blue light.

“We are excited about the enhanced benefits of the MCO platform, which includes the Max Planck Institute for Biophysics’ CatCh technology. “It offers new hope for restoring vision in people suffering from severe degenerative retinal diseases,” said Samarendra Mohanty, co-founder and president of Nanoscope Therapeutics. The Company is a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and geographic atrophy resulting from macular degeneration. “Our agreement with Max Planck Innovation enables us to translate scientific discoveries into effective therapeutic solutions. The MCO-010 fusion protein is bioengineered and administered using gene ferries to treat patients with severe degenerative retinal diseases. This puts us on the threshold of treating patients with high unmet needs and sustainably improving their quality of life.”

Mareike Göritz, patent and licensing manager at Max Planck Innovation, added: “The properties of CatCh combined with Nanoscope’s proprietary technology make this a very promising approach for the gene therapy treatment of retinitis-related visual impairment. We are excited about further developments and would be happy if patients ultimately benefit from this innovative approach.”

Nanoscope recently announced the 100 week data the multicenter, randomized, double-masked, sham-controlled Phase 2b/3 RESTORE clinical trial for retinitis pigmentosa (NCT04945772) known in the USA. The company also recently completed the Phase 2 STARLIGHT trial for the treatment of MCO-010 in patients with Stargardt’s disease (NCT05417126). MCO-010 has received Fast Track Designation and Orphan Drug Designation from the FDA for both retinitis pigmentosa and Stargardt disease. Preclinical products include the non-viral laser gene therapy MCO-020 for the treatment of GA secondary to age-related macular degeneration.