Clinical trial design and protocol development sit at the foundation of every successful study. The choices made at this stage, from study type and endpoint selection to patient stratification and engagement models, determine whether a program can recruit effectively, generate reliable data, and reach meaningful conclusions.
Genetics has become a defining factor in how clinical trials are designed, who qualifies, and how enrollment is planned. Genetic variation influences both disease development and treatment response, which means that recruiting, screening, and selecting participants for genetically stratified trials requires a fundamentally different approach to enrollment planning.
Last month, our team had the pleasure of attending the European Network to Cure ALS (ENCALS) meeting, an annual gathering that brings together some of the brightest minds in the field of amyotrophic lateral sclerosis (ALS) research and treatment. Each year, the ENCALS meeting reaffirms the commitment of the scientific community to ending ALS and brings renewed hope to patients and their families. While our team enjoyed networking with attendees and catching up on the latest research in ALS, this year they were also focused on Sano’s poster presentation for an initiative we are calling Light the Way.
£330K grant awarded to support and engage patients at risk of MND/ALS, and track the psychological impact of genetic testing.
This webinar, featuring Sano Genetics team members Ross Rounsevell and George Bowley, highlights the importance of purposeful participant engagement in research studies. The discussion centres around meaningful research participant engagement, its significance in research, strategies for effective engagement, and the future developments enabling better study design.
Our latest insights report focuses on Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder with significant implications for lung and liver health. AATD arises from a deficiency of the alpha-1 antitrypsin protein, which safeguards the lungs from damage. However, with this protein in short supply, the lungs become susceptible to inflammation, potentially leading to conditions like emphysema and chronic obstructive pulmonary disease (COPD).
This webinar, hosted by Paul Wicks and featuring a panel of experts in ALS and genetics, focuses on the challenges and variability in genetic testing and counselling for ALS. The experts highlight that while genetic testing is a critical component of ALS research and treatment, key challenges include making the test available to everyone who needs it and ensuring consistency among tests.
Mutations in the SOD1 gene were the first identified genetic cause of ALS, discovered in 1993. Yet SOD1-ALS accounts for only approximately 2% of all ALS cases, making variant-level research essential for understanding how this small but genetically defined population experiences disease onset, progression, and survival.
Clinical trials play a crucial role in the development of pharmaceutical and biotech products. However, unforeseen challenges can arise during the trial process, potentially jeopardising the integrity and outcomes of the study. As a recent Applied Clinical Trial article noted, “while drug discovery is mostly about science, drug development is rich with, and risked by, human drama.”
Data security is the practice of protecting digital information from unauthorized access, corruption, or theft throughout its entire lifecycle. For biotech companies working in genetics, this challenge is especially acute. Sensitive data, including genomic sequences, patient health information, and clinical trial records, moves across platforms, devices, cloud environments, and third-party vendor systems. Protecting that data at every stage is not optional. It is foundational to responsible research and long-term operational viability.