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Biotechs Apply Crispr Gene-Editing for Hemophilia

Crispr-Cas9 illustration

Crispr-Cas9 illustration (

1 June 2020. A treatment for the blood disease hemophilia and other inherited disorders are being developed with the gene-editing technology Crispr in a biotechnology deal. The collaboration based on Crispr technology developed at Intellia Therapeutics is bringing the Cambridge, Massachusetts company $100 million in an initial payment and equity stake from Regeneron Pharmaceuticals in Tarrytown, New York.

Crispr, short for clustered, regularly interspaced short palindromic repeats, 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 the editing enzyme, known as Crispr-associated protein 9 or Cas9, to specific genes needing changes.

Intellia is developing therapies with Crispr-Cas9 that remove disease-causing genes or mutations, repair genes with mutations found in small regions of DNA, and insert corrected or functioning genes when mutations are found in larger DNA regions. The company says its treatments are being designed to be either administered directly to patients, or delivered to cells taken from the patient, then cultured outside the body and transplanted back.

In the new agreement with biopharmaceutical developer Regeneron, the companies are applying the Intellia Crispr-Cas9 process to hemophilia, an inherited disorder where blood does not clot adequately, resulting in spontaneous or uncontrolled bleeding. In hemophilia, a genetic defect fails to produce proteins needed to mix with platelets allowing blood to coagulate. About 8 in 10 people with the condition have hemophilia type A, where the protein clotting factor 8 is missing. In type B, clotting factor 9 is missing. The companies report their preclinical research shows edited genes can produce normal or higher levels of factor 9 proteins in monkeys induced with hemophilia B.

Regeneron also gains non-exclusive rights to develop gene-editing treatments cultured outside the body and transplanted back in the patient. In addition, Regeneron receives rights to develop five more therapies that work in the liver, and 10 other disease targets outside the liver. Intellia receives an initial payment of $70 million, and an equity stake from Regeneron of $30 million, with the current agreement extended to 2024. Intellia is also eligible for further milestone payments and royalties.

The deal expands on the companies’ current collaboration begun in April 2016. In that agreement, Intellia and Regeneron are designing a treatment with Crispr-Cas9 for the inherited disease transthyretin amyloidosis, a rare disorder caused by a mutation that interrupts instructions to produce in the liver a protein called transthyretin. Without transthyretin, another protein called amyloid builds up on organs, tissues, and nerves in the body, affecting functions of the peripheral nervous system and other organs. The initial six-year deal enables Regeneron to discover up to nine more disease targets in the liver and other organs, with Intellia receiving $125 million in fees, milestone payments, and an equity stake from Regeneron.

“We believe that our Crispr-Cas9-based technology,” says Intellia president John Leonard in a statement, “addresses the limitations of current replacement and gene therapy approaches, and importantly, may provide a durable, potentially life-long solution to these genetic diseases.”

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