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Apellis uses Beam’s base editing technology to expand the scope of autoimmune diseases

Apellis Pharmaceuticals recently received FDA approval for the first time. The drug treats rare blood diseases by hitting specific targets in the complement system, a family of proteins that help immune system cells. The company hopes to bring complement system drugs to other diseases in other parts of the body, and is turning to Beam Therapeutics’ gene editing technology.

On Wednesday, the two companies announced a Research partnership This will apply Beam’s base editing technology to an undisclosed complement system goal. Beam will handle pre-clinical studies on procedures for up to six genes in the complement system. These genes are found in organs including the eyes, liver, and brain. Apellis has the right to license each program, and if these rights are exercised, it will be responsible for clinical development.

Apellis, headquartered in Waltham, Massachusetts, will prepay $50 million to Beam and another $25 million on the first anniversary of the contract. The $75 million payment is described as a combination of advance payments and recent milestone payments. Beam, headquartered in Cambridge, Massachusetts, can choose to share the development and commercialization of one of the licensing programs equally.

“This collaboration allows us to combine our know-how and capabilities in base editing with Apellis’ expertise in targeted complement pathways to develop new drugs for diseases driven by complement biology,” Beam Chief Executive Officer Officer John Evans said in a prepared statement. “This also represents an important strategic move to explore opportunities to expand the application of base editing and to solve more biologically complex diseases for patients who need new treatment options.”

May, the FDA approves Apellis drug Empaveli for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), A disease in which the complement system attacks red blood cells, causing blood clots and organ damage. The goal of Empaveli is a complement system called C3. New partnerships can develop other therapies that prevent C3. But Apellis and Beam said that base editing can also extend the complement system targeted therapy to other proteins in the complement cascade.

Beam’s basic editing technology provides a More precise genome editingUnlike CRISPR editing, which causes double-strand breaks in DNA, base editing can change individual letters of the genome. The two companies said that the application of this precise editing method has the potential to permanently change the complement cascade to solve diseases caused by the dysfunction of the complement system. The Apellis/Beam alliance was launched four months later Beam announces the acquisition of Guide Therapeutics for USD 120 million, A start-up company whose technology has identified lipid nanoparticles that are most suitable for delivering genomic drugs to specific tissue types.

Apellis and Beam did not disclose the diseases they intend to treat. For each project licensed by Apellis under the partnership, Beam will be eligible for additional milestone payments, and if any of them enter the market, there will be sales royalties. The value of these additional milestone payments was not disclosed. The cooperation spans five years, but can be extended to two years on a project-by-year and project-by-year basis.

photo: Lex, Getty Images

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