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Apellis Targets Rare Diseases With Gene Editing
31 Mar
Summary
- Apellis develops novel treatments for rare diseases.
- EMPAVELI treats paroxysmal nocturnal hemoglobinuria.
- SYFOVRE targets geographic atrophy in AMD.
Apellis Pharmaceuticals is dedicated to discovering and commercializing novel therapies for diseases with significant unmet needs. The company currently offers EMPAVELI, a treatment for paroxysmal nocturnal hemoglobinuria, and SYFOVRE, which targets geographic atrophy secondary to age-related macular degeneration.
Further development includes using EMPAVELI for C3 glomerulopathy, focal segmental glomerulosclerosis, and delayed graft function. Apellis is also developing APL-3007, an siRNA therapy for geographic atrophy, and APL-9099, a gene editing therapy targeting the neonatal Fc receptor for potential one-time dosing in future indications.
Collaborations with Swedish Orphan Biovitrum and Beam Therapeutics underscore Apellis's commitment to advancing its pipeline through strategic partnerships. These efforts focus on developing innovative treatments for complement-driven diseases, utilizing cutting-edge technologies like base editing.
