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Gene therapies for disorders such as X-linked Severe Combined Immunodeficiency (SCID, sometimes known as “bubble boy disease”), and Spinal Muscular Atrophy (SMA) have, for the first time, shown remarkable safety and efficacy results in clinical trials.
FDA approved Trikafta, a gene-based therapy creating hope for children with cystic fibrosis and Zolgensma, a gene therapy drug, are creating hope for children with spinal muscular atrophy.
On the diagnostic side, two examples for early stage cancer detection include:
- Significant progress with the 12 multi-cancer early blood detection test, and its FDA Breakthrough Device Designation (GRAIL).
- A liquid biopsy test proved being reliable as tissue-biopsy in lung cancer (Guardant360).
Research results were also impressive:
- Single-cell proteomics atlas of the tumor and immune ecosystem of human breast cancer reveals tumor and immune cell diversity
- Microbial gene diversity in the gut and oral human microbiome has been uncovered
- Lou Gehrig’s disease key gene has been identified
- Blood test aiming to detect breast cancer up to five years before symptoms has been postulated
- Comprehensive genomic tumor profiling to identify tumor driver genes has been demonstrated in a multi cancer study
Finally, and very importantly was the move of genome sequencing into clinical care.
Just a couple of examples include:
- Apple to integrate Allscripts’ EHR data into its Health Records platform
- Release of 50,000 individual’s UK Biobank genetic data, generated via the UK Biobank, Regeneron, and GSK collaboration, as a resource to the community.
We should be very proud of these 2019 clinical plan and development advances!
We should continue to look at the bright future ahead of us with the commitment to create clinical plans for the development of new therapies and study designs that will enable us to bring a long, bright future to patients.
Specializing in rare disease, Boston Biotech Clinical Research works with biotech, pharmaceutical, device companies and investors to streamline the clinical trial process. Our experienced team helps each client reach their specific goals by customizing a clinical and regulatory road map of simplified programs and streamlined protocols to meet our clients’ requirements.