Alnylam, a biopharmaceutical company, has presented promising initial results from its investigational gene silencing therapy, which indicates that it can largely eliminate a key protein involved in Alzheimer's disease. Alnylam's small interfering ribonucleic acid (RNAi) molecule, named ALN-APP, was shown to eliminate or knock down the gene responsible for producing the amyloid precursor protein (APP), which causes early onset Alzheimer's disease via amyloid plaques formed in the brain and associated with neurodegeneration. In a Phase I study, 20 patients treated in three single-dose cohorts saw a dose-dependent, rapid, and sustained reduction of up to 90% in APP production, assessed by biomarkers in cerebrospinal fluid, with response sustained for at least three months. Alnylam's CEO Yvonne Greenstreet called the results "really groundbreaking" and confirmed the company's intention to proceed with Part B of the study, despite the US Food and Drug Administration's (FDA) partial clinical hold on the multi-dose study in the US. The FDA has placed a partial clinical hold on the multi-dose Part B study due to findings observed in prior non-clinical chronic toxicology studies.
About Alnylam Pharmaceuticals
Alnylam Pharmaceuticals is a biopharmaceutical company focused on RNA interference (RNAi) therapeutics for genetically defined diseases. Its RNAi platform allows for the development of drugs that can silence or "turn off" specific genes that cause diseases. Alnylam has several approved drugs targeting liver diseases, such as Onpattro for the treatment of hereditary transthyretin-mediated amyloidosis.
The company has also been working on expanding the use of its RNAi platform for CNS diseases, including Alzheimer's and Huntington's diseases, through a partnership with Regeneron. Alnylam's recent early stage data on a gene silencing therapy targeting Alzheimer's disease has shown promising results.
As of May 2023, Alnylam has a market capitalization of approximately $30 billion and is headquartered in Cambridge, Massachusetts. The company has collaborations with various pharmaceutical companies and academic institutions to advance the development of RNAi therapeutics.
Alnylam Pharmaceuticals, Inc. is a publicly-traded company with shares listed on the NASDAQ stock exchange under the ticker symbol "ALNY"
Reference
Alnylam Buoyed By Early Alzheimer’s Gene Silencing Success