Excellence in research & development
Neurology research at Biogen is focused on identifying novel therapeutic approaches for devastating neurological and neurodegenerative conditions such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis and multiple sclerosis, aiming to move drug candidates into human clinical testing as safely, quickly and efficiently as possible.
Internally and with our many academic partners, we have a major focus on target discovery and validation using human stem cell models, functional cell-based screening, gene network and expression analysis, and study of disease-resistant neuronal populations. We functionally validate candidate targets in the most relevant models and seek evidence of their involvement in human disease.
Subsequently, we explore with our colleagues across Biogen the therapeutic modality – small molecules, antisense oligonucleotides, antibodies or biologics – best suited to the patient population we are seeking to help. Our aim for each target is to create a complete package of information based on high-level mechanistic biology and rigorous testing that will give the program the best chance of success as it moves toward the clinic.
At Biogen, neuroimmunology research concentrates on understanding mechanisms by which irreplaceable neurons and other central nervous system (CNS) cells are injured or destroyed during the course of neurodegenerative diseases including Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS) and multiple sclerosis. We emphasize how these diseases alter the CNS environment and thereby promote disease. We study microglia, astrocytes and the blood-brain barrier, elements whose function is essential for neuronal health. Our research is directly complementary and integrated with that of the Neurology and Pathway Discovery groups.
Our research targets are selected by virtue of their genetic validation in neurodegenerative disease. Genetic studies done within Biogen and with our academic collaborators direct us toward the most salient targets and allow us to understand how pathogenic genetic variants alter protein function or expression, leading to effects on cells, which produce downstream disease-promoting processes. The technologies brought to bear at this stage of research include high resolution microscopy and imaging, flow cytometry, gene expression and epigenetic profiling, behavioral assessment and neuropathological studies.
The hematology area at Biogen has grown out of our core hemophilia research group that discovered the recombinant clotting factor VIII and IX monomeric Fc fusion proteins and advanced them into clinical development for the improved treatment of hemophilia A and B, respectively. Moving beyond our initial assessment of these molecules, our scientists continue to advance our knowledge of their structure and activity relationships, as well as to better understand their mechanisms of action for half-life extension and generating tolerance/immunogenicity.
Our group continues to explore novel therapies for the treatment of hemophilia, with an initial focus on further increasing the half-life of FVIII, and creating innovative therapies for people with hemophilia A and B who have developed neutralizing antibodies (inhibitors) against their current treatment options. We are now further expanding our research into related areas of nonmalignant hematology, such as sickle cell disease.
In all of our work, we seek to apply a combination of advanced techniques with pharmacology studies in relevant models to further understand the physiological interactions in these disease areas and better guide our protein engineering and drug development research. Our active collaborations with both academic and industry groups are essential. They enable us to apply the most advanced technologies and encompass the widest range of ideas to achieve our goals for transforming care of hematologic diseases with high unmet medical needs.
Convergence Pharmaceuticals, a Biogen company based in the United Kingdom, has world-class expertise in new treatment approaches to chronic pain.
The goal of analgesic treatment in addressing chronic pain is to suppress the hyperexcitability in a patient’s pain signaling network and to set the network activity back to the levels experienced before injury. We believe that the best strategy to achieve this goal is to target the points of convergence in pain signaling. Our research targets ion channels, specifically sodium channels (Nav), involved in the generation and propagation of electrical signals in nerve cells.
Convergence research has developed a leading pipeline of small molecules that have the potential to selectivity target the high frequency firing of signals associated with chronic, neuropathic pain while sparing the lower frequencies of firing associated with normal functions such as cardiac activity.
Convergence’s in-house technology platform is powered by world-class capabilities in ion channel electrophysiology and ion channel medicinal chemistry. Our chemistry experience combines a mastery of complex synthesis with extensive know-how ranging from hit identification through clinical candidate selection. We are exploiting recent advances in high throughput electrophysiology, which together with sensory nerve and spinal cord electrophysiology may enable the development of new differentiated pain medicines.
Cell & Gene Therapy
Biogen’s Cell & Gene Therapy team is dedicated to becoming a leader in bringing new treatments to market and contributing to the continued evolution of the field.
Our strategy is to collaborate with leaders in academia and industry who represent a range of gene therapy, genome editing, and delivery technologies. While actively seeking collaborations, we are building an industry-leading internal capability that can apply a variety of approaches to therapeutic challenges.
Biogen is currently engaged in gene therapy programs in ophthalmology, hemophilia A and B, sickle cell disease and beta thalassemia. We have one program for X-linked Retinoschisis (XLRS), a rare eye disorder, in the clinic. Biogen intends to pursue opportunities across our therapeutic focus areas where we believe there is an opportunity to significantly improve the lives of patients.
The Cell & Gene Therapy team is fully integrated into Biogen, enabling us to take a holistic approach to developing potential therapies by applying our expertise in areas such as research and development, manufacturing and regulatory affairs.
Epigenetic mechanisms mediate the impacts of cellular and organismal life histories – within and between generations – on implementation of the instructions for life that are encoded within our genomes. They encompass those mechanisms that modulate gene function without altering gene sequence. Our recognition of the complexities of epigenetic regulation – mediated by DNA and histone modifications, and by noncoding RNAs – is growing in parallel with our understanding of the mechanisms by which epigenetic dysregulation leads to cell dysfunction and disease, and our hope that epigenetic modulation of genetic, biochemical and cellular functions will be the basis for new therapies for the treatment of many human diseases.
The epigenetics group at Biogen focuses on understanding epigenetic mechanisms that operate in normal and diseased cell states, with the intention of leveraging epigenetic biology to develop new therapies for degenerative diseases in the areas of neurology, immunology, hematology, tissue injury and fibrosis. We pursue this work using the concepts and tools of cell biology, biochemistry, proteomics, genetics, epigenetics, genomics, epigenomics and computational biology. Our pursuit of insights into epigenetic mechanisms in health and disease is grounded in molecular interactomics, cell-based screening along the epigenetic axis and network analysis. We are utilizing these approaches to garner new insights into epigenetic biology in normal and diseased cell states, and leveraging those insights for the development of new treatments that ameliorate or prevent degenerative disease.
Biogen is committed to understanding the genetic basis of the diseases on which we focus.
As part of that commitment, our Precision Medicine group applies whole genome and exome sequencing, genomics and computational biology throughout the drug discovery and development process. Our mission is to find the right targets, the right biomarkers, and the right patients: We conduct work in-house and with leading academic institutions to identify new genes implicated in disease, drive the discovery of novel targets to fuel our drug discovery efforts, and potentially stratify patients for our clinical trials. In our quest to gain new insights into disease, we also apply systems biology using computational methods to analyze genes and pathways.
The Precision Medicine team also uses these technologies to segregate clinical trial biomarker data to look for molecular signatures that are predictive of disease severity and outcomes, potentially driving new treatment approaches and strategies.
Cell & Protein Sciences
The Cell & Protein Sciences team develops protein and imaging tools to explore novel biological pathways and designs and then engineers and optimizes therapeutic drug candidates for clinical development. We partner closely with the biology areas and apply innovative protein and in vivo science approaches.
Our primary research interests are in the following areas:
- Antibody discovery and engineering
- Protein engineering, expression and characterization
- Preclinical imaging including PET, SPECT, MRI, optical and ultrasound
- Technologies including CRISPR- and TALEN-based methods for genome engineering
Chemistry & DMPK
The mission for the Chemistry & DMPK (Drug Metabolism and Pharmacokinetics) team at Biogen is to identify novel and differentiated oral small molecule drug candidates for the treatment of diseases of interest and move these candidates into human clinical testing. We do this in close partnership with biologists, pharmacologists, toxicologists and others.
This group utilizes high-throughput and phenotypic screening to discover chemical leads. The leads are optimized for potency, selectivity, cellular activity and ADME/PK properties via iterative cycles of structure-based drug design, hypothesis-driven approach and parallel organic synthesis. We work with preclinical imaging and chemoproteomics groups in designing probes to evaluate the target engagement of our clinical candidates.
Biogen's growing small-molecule drug-discovery group will enhance the pipeline by expanding the drug target pool and pharmacologic treatment modes accessible to protein therapeutics.