CRISPR Medicine

Apellis Pharmaceuticals

Company Type: Therapeutic development

Main focus: Developing novel therapeutics through targeting of complement protein C3

Company stage: Clinical (pre-clinical for gene editing)

Diseases: Liver, retinal, and neurological diseases

Genome-editing tool: Base editing

Funding stage: Post-IPO Debt

Location: Waltham, Massachusetts, USA

Website: https://apellis.com/

Pipeline: https://apellis.com/our-science/our-pipeline/

Partners: Beam Therapeutics


Apellis Pharmaceuticals is a biopharmaceutical company developing targeted therapies for various diseases. The company focuses on inhibiting the complement system via complement protein C3 targeting. Apellis’ broad portfolio includes approved non-gene-editing therapies for the rare disease paroxysmal nocturnal hemoglobinuria and the eye disease geographic atrophy. Apellis recently entered an exclusive collaboration with Beam Therapeutics to develop an undisclosed gene-editing programme.

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