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Company Started for Gene Editing Rare Inherited Diseases

Mitochondria illustration

Mitochondria (Charlie Jones, Pixabay.

30 Dec. 2022. A new enterprise is formed to develop gene-editing therapies for mitochondrial disorders, rare inherited mutations in the energy centers of cells. The new company — Primera Therapeutics Inc. in Rockville, Maryland — is spun off from Mayflower Bioventures, a gene and cell therapy start-up incubator, and adapting its gene-editing technology from Cellectis SA, a biotechnology company in Paris.

Primera Therapeutics aims to develop treatments for inherited diseases of the mitochondria, components of cells that convert oxygen and nutrients into the chemical adenosine triphosphate, the provider of energy for metabolism in cells. Mitochondrial diseases are chronic, long-term inherited disorders resulting from mitochondria that fail to provide sufficient energy for cells. While these diseases can occur at any age, in many cases they appear in infancy, and can affect cells in any parts of the body. Cleveland Clinic says one in 5,000 people have a genetic mitochondrial disease, with 1,000 to 4,000 children born each year in the U.S. with the condition.

Primera Therapeutics is the first new business started by Mayflower Bioventures, a joint venture of Hibiscus BioVentures, a venture capital and business development company in Rockville, Maryland, and the Mayo Clinic, to commercialize discoveries at Mayo Clinic’s regenerative medicine labs. Science & Enterprise reported on formation of Mayflower Bioventures in Aug. 2022. The company’s process is based on gene-editing research in Mayo Clinic labs by geneticists Stephen Ekker and Karl Clark. Ekker is the scientific founder of Primera Therapeutics.

Collaboration and option for licensing

For mitochondrial disease therapies, Primera plans to use a gene-editing technology called transcription activator-like effector nucleases or TALENs. Nucleases are enzymes that can cleave DNA, and can be designed to target precise locations in DNA. TALENs is an earlier form of genome editing that pre-dates Crispr, a gene-editing technique whose developers won the Nobel Prize in chemistry in 2020. The company plans to apply TALENs gene editing to repair disease-causing mitochondrial DNA.

Primera Therapeutics is adapting the TALENs technology developed by the biotechnology company Cellectis. According to Cellectis, its TALEN process uses editing nuclease enzymes that bind to specific DNA locations, within 33 or 34 amino acid positions in DNA chemistry. Primera and Cellectis are collaborating on creating genome editing tools for mitochondrial DNA with TALENs. Under their agreement, Primera can exercise an option for a worldwide exclusive license to the TALENs process for up to five products developed as part of the collaboration.

Should Primera exercise this option, Cellectis would be eligible for up to $750 million in development and sales milestone payments. Cellectis and Primera Therapeutics are considering other undisclosed arrangements beyond this first collaboration, which if concluded, would give Cellectis a 19 percent equity stake in Primera as well as a seat on the company’s board.

Primera says it’s also collaborating with United Mitochondrial Disease Foundation that promotes awareness and research on mitochondrial diseases, to encourage new diagnostics and treatments. The group says it supports research on mitochondrial diseases at 125 labs, generating another $150 million in government research funding.

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