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Apellis and Beam Collaborate to Apply Base Editing for Complement-Driven Diseases Drug Discovery

July 1, 2021

Apellis Pharmaceuticals and Beam Therapeutics entered an exclusive five-year research collaboration focused on the use of Beam’s proprietary base editing technology to discover new treatments for complement-driven diseases.

Photo: Cedric Francois, co-founder and chief executive officer, Apellis

The complement system is a part of the immune system that enhances its ability to clear microbes and damaged cells from an organism, promote inflammation, and attack the pathogen’s cell membrane. The collaboration will combine Apellis’ expertise in complement, a complex biological system, with Beam’s proprietary base editing platform on six research programs focused on C3 and other complement targets in the eye, liver, and brain.

“Beam has pioneered base editing, which holds significant promise as a best-in-class technology for precision gene editing. This collaboration builds on our deep scientific expertise in complement and, together with our growing pipeline, positions Apellis for long-term leadership in the complement field,” said Cedric Francois, co-founder and chief executive officer, Apellis.

Base editing represents a potential new class of precision genetic medicine that uses a chemical reaction designed to create precise, predictable, and efficient single base changes at targeted genomic sequences without making double-stranded breaks in the DNA. Editing key elements of the complement pathway in target organs has the potential to alter the complement cascade and durably address diseases driven by abnormal complement activity.

“Apellis has established itself as a leader in complement with the advancement of compelling targeted C3 therapies,” said John Evans, chief executive officer, Beam. “This collaboration allows us to combine our proprietary technologies and capabilities in base editing with Apellis’ expertise in targeting the complement pathway to develop new medicines for diseases driven by complement biology. This also represents an important strategic initiative to explore opportunities that expand the application of base editing to address more biologically complex diseases for patients in need of new treatment options.”

Under the terms of the collaboration agreement, Beam will apply its base editing technology and conduct preclinical research on up to six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis will have exclusive rights to license each of the six programs and will assume responsibility for subsequent development. Beam may elect to enter a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.

As part of the collaboration, Beam will receive a total of $75 million in upfront and near-term milestones from Apellis, which includes $50 million upon signing and an additional $25 million payment on the one-year anniversary of the contract execution date. After exercise of the opt-in license rights for each of the up to six programs, Beam will be eligible to receive development, regulatory, and sales milestones from Apellis, as well as royalty payments on sales. The collaboration has an initial term of five years and may be extended up to two years on a per year and program-by-program basis.

Author: Rare Daily Staff

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