ALNY
- Alnylam Pharmaceuticals, Inc.
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Overview
Company Summary
Alnylam Pharmaceuticals, Inc. (ALNY) is a biopharmaceutical company specializing in the development and commercialization of innovative RNA interference (RNAi) therapeutics.
RNAi is a naturally occurring biological process that regulates gene expression, and Alnylam focuses on harnessing this process to develop novel drugs for the treatment of debilitating diseases. The company's core technology platform, called RNAi therapeutics, allows for the selective silencing of disease-causing genes, thereby potentially providing a new approach to treating a wide range of diseases that were previously considered untreatable.
Alnylam's pipeline includes a diverse portfolio of investigational RNAi therapeutics targeting various diseases, such as genetic and rare diseases, cardiovascular diseases, hepatic infectious diseases, and cancers. They collaborate with leading academic institutions, biotechnology companies, and pharmaceutical partners to accelerate the development and commercialization of these potential treatments.
The company has also invested in developing its own delivery technologies to efficiently and safely deliver RNAi therapeutics to specific tissues and cell types in the body. This includes utilizing lipid nanoparticles and conjugate-based systems for targeted delivery, allowing for improved drug efficacy and patient outcomes.
Alnylam has received regulatory approvals for some of its RNAi therapeutics, such as ONPATTRO� (patisiran), which is used in the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. They also have several other candidates in late-stage clinical development and multiple programs in early-stage clinical and preclinical studies.
By leveraging the potential of RNAi therapeutics, Alnylam Pharmaceuticals aims to provide transformational therapies that could address the unmet medical needs of patients suffering from various diseases, ultimately improving their quality of life.