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Biotech Creating More Efficient Crispr Therapies

DNA puzzle

(Arek Socha, Pixabay)

30 Nov. 2020. A biotechnology company is developing treatments for cancer and other diseases using Crispr gene editing delivered with more efficient synthetic proteins. Spotlight Therapeutics Inc., a three-year-old enterprise in Hayward, California, is emerging from stealth operations and raising $30 million in its first venture funding round.

Spotlight Therapeutics says it’s creating Crispr gene editing therapies that work more precisely and efficiently than current methods. Crispr, short for clustered regularly interspaced short palindromic repeats, is a genome-editing process based on bacterial defense mechanisms that use RNA to identify, monitor, and edit targeted locations in DNA. The company says therapies based on Crispr, such as for inherited diseases, are delivered today with benign viruses or in lipid nanoscale particles that limit their full potential.

Spotlight’s platform designs programmable synthetic proteins that deliver and carry out Crispr gene editing inside targeted cells. The company’s technology is based on research by its scientific founders, geneticists Alex Marson at University of California in San Francisco, Jacob Corn at ETH Zurich in Switzerland, and Patrick Hsu at University of California in Berkeley. The founders’ labs study biologic processes that call for enhancing and refining earlier forms of gene editing with Crispr. Marson, Corn, and Hsu are company directors and scientific advisors.

Spotlight calls its platform targeted active gene editors that assemble different molecular components to deliver and perform Crispr gene editing. The company says it maintains a library of these components, including peptides that permeate cell membranes, ligands or binding molecules, and antibodies. Spotlight then puts together these components in a package with gene-editing enzymes that maximizes targeting and delivery of Crispr functions.

“Spotlight’s unique approach represents the potential to expand applications and democratize gene editing for patients,” says Marson in company statement.

The company’s first treatments address cancer and hemoglobin disorders, such as sickle cell disease. For treating cancer, Spotlight Therapeutics is building a targeted active gene editor that edits T-cells and macrophage cells in the immune system for altering the cancer tumor microenvironment. That microenvironment of blood vessels and extracellular material, supports cancer cell growth and protects the tumor against immune system responses. Spotlight says its cancer therapies aim to weaken the microenvironment, making it less resistant to immune responses. The company is beginning with treatments injected directly into tumors, but says it plans to ultimately develop a systemic therapy.

For hemoglobin diseases, Spotlight is developing a targeted active gene editor that edits blood-forming stem cells in bone marrow. Hemoglobin is a protein in blood that delivers oxygen to cells in the body. Current Crispr treatments in clinical trials for sickle cell disease first require editing a patient’s blood-forming stem cells outside the body, then reinfusing them back into the patient. Spotlight plans to develop a Crispr therapy that edits blood-forming stem cells while in bone marrow, making the process much simpler and safer.

The company is raising $30 million in its first venture round, led by GV, formerly Google Ventures, and joined by other undisclosed investors. According to Crunchbase, Spotlight Therapeutics already raised $14.2 million beginning in January 2018, thus some or all of that amount may be included in the $30 million figure.

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Hat tip: Endpoints News

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