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Eye Disease Gene-Therapy Biotech Adds Venture Funds

Monitor eyes

(Gerd Altmann, Pixabay)

15 Sept. 2022. A biotechnology company designing treatments for inherited eye diseases with Crispr gene editing is raising €75 million in venture financing. SparingVision, a six year-old business in Paris, is developing genetic therapies for retinitis pigmentosa, other inherited retinal disorders (IRDs), and dry age-related macular degeneration (AMD).

Retinitis pigmentosa or RP is a group of rare diseases of the retina, the light-sensitive tissue at the back of the eye. RP is a progressive disorder, where retina cells degrade and breakdown leading increasing loss of vision over time. According to National Eye Institute, RP symptoms often start in childhood, beginning with loss of night vision. People with RP usually experience a narrowing field of vision, losing peripheral vision, then eventually loss of central vision and blindness. SparingVision cites data showing some 2 million people affected by RP worldwide, including 40,000 in France.

SparingVision designs gene therapies delivered to the retina to treat inherited eye diseases. The company’s lead product, code-named SPVN06, delivers two small proteins called neurotrophic factors naturally produced by photoreceptor cells in the retina, expected to protect remaining photoreceptors and prevent further retinal degradation. The company says SPVN06 is in preclinical testing.

Deliver Cas9 enzymes to remove disease-causing mutations

In Oct. 2021, as reported in Science & Enterprise, the company began a collaboration with the biotech enterprise Intellia Therapeutics, a developer of genetic treatments using the gene editing technology Crispr, short for clustered, regularly interspaced short palindromic repeats. Crispr is adapted from a natural process used by bacteria to protect against attack by viruses, where an enzyme that deactivates or replaces genes binds to targeted RNA molecules generated by the genome. The RNA molecules then guide an editing enzyme, known as Crispr-associated protein 9 or Cas9, to specific genes needing changes.

SparingVision says it uses adeno-associated viruses, or AAVs, to deliver gene therapies to the retina. These viruses are benign, naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, and generate at most a mild immune response. With Crispr, the company says AAVs deliver Cas9 enzymes to remove disease-causing mutations in retinal cells, after which AAVs deactivate. SparingVision says its work with Intellia also includes investigating genetic therapy delivery with nanoscale lipid or natural oil particles, a technique used with Covid-19 vaccines.

The company is raising €75 million ($US 75 million) in its second venture funding round. Leading the financing are life science investor Jeita Capital in Paris and UPMC Enterprises in Pittsburgh. Taking part in the round are  4BIO Capital, Bpifrance, the RD fund — venture arm of Foundation Fighting Blindness — and Ysios Capital. Proceeds from the round are expected to advance SPVN06 to clinical trials, as well as its other gene therapies in development. Crunchbase says the company so far raised a total of $127.6 million.

“With this financing,” says SparingVision CEO Stéphane Boissel in a company statement, “we are taking a significant step towards achieving clinical validation of our two lead assets and bringing mutation-agnostic genomic medicines to millions of patients affected by IRDs and dry AMD.”

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