Preclinical feature articles
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Beyond Nuclear Genetics: Let's Think Differently About CNS Disease Targets
9/12/2022
Nuclear genes with mutations remain the primary focus for selecting biological drug targets, but we have yet to see a drug come to market from a genetically validated target that can arrest or reverse diseases like Parkinson’s. We need to expand our research to explore the essential drivers, genetic or otherwise, that switch our cells from order to disorder or from function to dysfunction.
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Your 3-Step Process For Innovation In Precision Medicine
8/3/2022
Scientific advances are exciting, but not every discovery translates well into the clinic. Why is that? And how can companies successfully position themselves for market success with their drug discovery efforts?
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Uncovering Rare Pediatric Diseases Through Atypical Gene Associations
5/12/2022
A conversation with Carol Saunders, PhD, FACMG, FABMGG, and Isabelle Thiffault, PhD, FABMGG, illuminates the genome sequencing research work they are doing at the Clinical Genetics and Genomics Laboratory at Children's Mercy Kansas City and the Genomic Medical Center at Children's Mercy Research Institute.
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Shining Light on the Commercial Potential of Optogenetic Therapies
2/3/2022
Light can be used to stimulate light-sensitive receptors in order to turn brain cells “on” and “off” at the flip of a switch, called optogenetics. Until recently, its use has been restricted to use in preclinical animal models, but now, advances have allowed this technology to move from the lab to humans. This article shares recent advances in optogenetics, the landscape of optogenetic therapies currently in clinical trials, and more.
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Sex Matters… In Preclinical Drug Development?
1/18/2022
In safety pharmacology studies investigating short-term side effects on physiological functions, the scientist is obliged to “consider” sex in the study design of animal models. This article also shares the differences in anxiety and depression in human males and females.
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Genome Sequencing Research For Rare Diseases & The Future Of The Field
1/11/2022
Tomi Pastinen, M.D., Ph.D., is the director of the Genomic Medicine Center at Children’s Mercy Research Institute. In 2019, he and a team of CMRI researchers launched Genomic Answers for Kids, a first-of-its-kind pediatric data repository with the goal of collecting genomic data and health information from children and their families over the next seven years to create a database of 100,000 genomes.
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Can Anti-TNF Treat Post-operative Cognitive Decline? New Clinical Research Investigates
1/6/2022
People who suffer from post-operative cognitive decline can develop difficulty remembering things and performing daily tasks, and may possibly end up in nursing homes. There are no existing therapies. However, 180 Life Sciences believes it has a novel understanding of how to treat it.
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Harnessing The Human Virome For The Next Generation Of Gene Therapy Vectors
1/5/2022
Advances in metagenomic sequencing have revealed insights into the human virome with boundless value for gene therapy researchers. This includes exploring the diversity of a family of small commensal viruses called anelloviruses that have never been associated with disease. Anelloviruses are a promising new tool in our effort to overcome today’s limitations in genetic medicine.
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Why Every Biotech Needs Qualified Drug Hunters
11/29/2021
Christopher Kirk, co-founder, president, and CSO of Kezar Life Sciences, believes every biotech needs a good drug hunter. “This is an industry that is rife with failure,” says Kirk. “Every failure will cost companies a lot of time and money. Every company needs their scientists focused on meaningful drug discovery research with the goal of reducing this failure rate.”
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6 Key Steps For Cell Therapy Clinical Development Success
9/14/2021
While the simplest option for a small or emerging biotech is to outsource protocol and clinical development to a consultant or CRO, a disengaged or absent sponsor is unlikely to have its objectives realized. Prior to engaging with a consultant or CRO, you can take these six steps that are likely to leave both parties engaged in the clinical development process.