In our latest report, we explore the complexities of Amyotrophic Lateral Sclerosis (ALS), offering a comprehensive overview of the disease. From genetic components to the latest FDA-approved treatments, we cover the entire spectrum of ALS research and care.
Metabolic dysfunction-associated steatohepatitis (MASH), previously known as nonalcoholic steatohepatitis (NASH), is a complex liver disease characterized by fat accumulation, inflammation, and fibrosis. With its global prevalence rising alongside obesity and type 2 diabetes rates, the medical community is urgently seeking innovative prediction, diagnosis, and treatment strategies.
This webinar, hosted by Sano CEO Dr. Patrick Short and featuring David Ochoa, Platform Coordinator at Open Targets, explores the significance of human genetics in pinpointing drug targets, the variety of data sets employed, target safety, and methods of intervention. It also touches on the changing nature of drug modalities and prospective avenues for future research.
As precision medicine trials increasingly depend on genetic eligibility criteria, the process of testing, interpreting results, and guiding patients through their implications becomes a central part of study execution. Genetic counselors serve as a critical resource of information about genetic disorders for healthcare professionals, patients, and the general public, translating complex genomic data into actionable understanding.
Genetic testing has become a defining capability in biotechnology and pharmaceutical development. By revealing the specific genetic variants that influence disease risk and drug response, it enables a more targeted approach to both treatment design and clinical research. Its applications now span oncology, rare disease, pharmacogenomics, and preventive health, reshaping how therapies are developed, tested, and delivered.
Digital twins are virtual models designed to accurately reflect a physical object or system. The concept comes from engineering and has been applied to complex systems such as airplanes, manufacturing, and even cities. While the use of digital twins in healthcare is still very new, it is already enabling more personalized treatments and creating a better understanding of patient health.
While lifestyle choices and environment can impact risk, genes are often what most affect how likely a person is to develop a disease. People commonly have different versions of a gene throughout a population, and some of these versions are associated with an increase in disease risk. Polygenic risk scores consider these gene variations and can provide a measurement of a person's risk for a specific disease.
For most clinical programs, recruitment is the single largest source of delay. It is often more operationally complex than the trial itself, and when it underperforms, the consequences extend well beyond the enrollment timeline. Protocols stall, costs compound, and treatments reach patients later than they should. The challenge is not simply finding patients. It is identifying the right patients, qualifying them efficiently, and maintaining their engagement across an increasingly complex study lifecycle.
CRISPR (clustered regularly interspaced short palindromic repeats) and CRISPR-associated (Cas) proteins were first characterized as part of bacterial immune defense and later adapted into programmable genome editing tools that enable site-specific DNA modification. This shift from microbial biology to clinical development has made CRISPR a central technology in precision medicine, including the first approved therapies for inherited blood disorders (NSF).
Metabolic dysfunction-associated steatohepatitis (MASH), previously known as nonalcoholic steatohepatitis (NASH), is a progressive liver disease driven by fat accumulation that triggers chronic inflammation and fibrosis. It represents the more severe end of the metabolic dysfunction-associated steatotic liver disease (MASLD) spectrum, which ranges from simple steatosis to advanced fibrosis, cirrhosis, and hepatocellular carcinoma. Without intervention, MASH can lead to irreversible liver damage.