Gene Silencing, Licensing, Production and Regulation

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam is hoping that revenue will stream from its new products and payments from Novartis, which owns the rights to cholesterol lowering drug inclisiran.

FDA Approval

FDA Approval Gene Silencing Drugs RNA

uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

Gene Therapy

Gene Therapy Licensing Licensing Gene

MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

pharmaphorum

JUNE 8, 2022

The UK medicines regulator has awarded Alnylam’s RNAi-based therapy zilebesiran for hypertension an ‘innovation passport ‘, a designation designed to speed up NHS access to promising new medicines.

Gene Silencing

Gene Silencing Drugs Medicine Regulation

Clinical Research Informer

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

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