Our Scientific Approach
At Alector, we are working relentlessly to change the course of neurodegenerative diseases.
Our goal is to remove toxic proteins, replace deficient proteins, and restore immune and nerve cell function. Drawing on insights from human genetics, neuroscience, and immunology, our scientific approach centers on three critical pillars: Unlocking Genetic Pathways, Enhancing Brain Delivery, and Advancing Biomarker-Guided Development. Together, these strategies form the foundation for our pursuit of transformative therapies that aim to slow or even halt the progression of frontotemporal dementia, Alzheimer’s, Parkinson’s, and other neurodegenerative diseases.
Unlocking Genetic Pathways
We believe that unlocking the genetic mechanisms behind neurodegenerative diseases is key to developing effective therapies. Our approach begins with an in depth understanding of human genetics, using data-driven insights to identify mutations and pathways that contribute to disease. By focusing on genetically validated targets, we are advancing approaches to counteract the underlying pathologies of these diseases.
Our research integrates advanced bioinformatics and human pathology to pinpoint critical genetic risk factors that drive neurodegeneration. Through techniques like CRISPR activation and inhibition, proteomics, and transcriptomics, we validate these targets and engineer therapeutic molecules designed to counteract the damage caused by these mutations. This approach allows us to pursue therapies that address the fundamental drivers of neurodegenerative diseases, not just the symptoms, providing hope for better patient outcomes.
Enhancing Brain Delivery
One of the greatest challenges in treating neurodegenerative diseases is delivering effective treatments across the blood-brain barrier. Our proprietary Alector Brain Carrier (ABC) technology is addressing this challenge. The blood-brain barrier serves as a protective mechanism for the brain, but it also restricts the delivery of many therapeutics. ABC is designed to enhance the brain penetration of therapeutic agents, enabling them to reach the brain in sufficient quantities and with the potency needed for efficacy.
ABC uses receptor-mediated transcytosis to cross the BBB, targeting specific receptors on endothelial cells to facilitate the delivery of therapeutics. This technology is not only designed to improve drug delivery to the brain but also aims to reduce efficacious dose levels, potentially broadening the therapeutic window and minimizing side effects. By optimizing drug delivery, we aim to enhance the effectiveness of treatments while lowering costs, paving the way for more accessible and efficient therapies.
Advancing Biomarker-Guided Development
The journey to effective treatments may be accelerated by the use of biomarkers – molecular indicators that help us track disease progression and therapy effectiveness. Biomarkers play a critical role in our drug development process, helping us advance therapies for neurodegenerative diseases. They provide valuable insights into disease progression, monitor target engagement, and assess therapeutic impact.
Diagnostic biomarkers guide our ability to select the right patient populations for our therapies, facilitating our efforts to run clinical trials that are more efficient and informative. Pharmacodynamic biomarkers further support this by helping us measure disease activity and track the effects of treatment on disease progression.
Biomarkers not only help in assessing therapeutic impact but also serve as valuable tools in discovering new drug targets, which can lead to the development of more effective and precise treatments. By combining biomarker data with clinical outcomes, we are able to refine our treatment strategies and enhance the precision of our drug development efforts.
This approach allows us to make more informed decisions throughout the drug development process, optimizing the likelihood of success and potentially accelerating the delivery of medicines to patients in need.