What rare diseases does Apellis Pharmaceuticals treat?

Apellis Pharmaceuticals develops treatments for rare diseases such as paroxysmal nocturnal hemoglobinuria and geographic atrophy secondary to age-related macular degeneration, as well as C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis.

What are EMPAVELI and SYFOVRE?

EMPAVELI is a treatment offered by Apellis for paroxysmal nocturnal hemoglobinuria, while SYFOVRE targets geographic atrophy related to age-related macular degeneration.

Is Apellis Pharmaceuticals developing gene editing therapies?

Yes, Apellis Pharmaceuticals is developing APL-9099, a gene editing treatment targeting the neonatal Fc receptor, with the potential for one-time dosing in future indications.

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 Targets Rare Diseases With Gene Editing

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.

Disclaimer: This story has been auto-aggregated and auto-summarised by a computer program. This story has not been edited or created by the Feedzop team.

Home / Health / Apellis Targets Rare Diseases With Gene Editing

Apellis Targets Rare Diseases With Gene Editing

31 Mar

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Summary

Apellis develops novel treatments for rare diseases.
EMPAVELI treats paroxysmal nocturnal hemoglobinuria.
SYFOVRE targets geographic atrophy in AMD.

Disclaimer: This story has been auto-aggregated and auto-summarised by a computer program. This story has not been edited or created by the Feedzop team.