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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics 189
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Hopewell Therapeutics raises funds for genomic medicines development

Pharmaceutical Technology

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

Genome 130
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STAT+: Moderna expands CRISPR gene editing research with ElevateBio partnership

STAT News

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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World's largest autism whole genome sequencing study reveals 134 autism-linked genes

Medical Xpress

Researchers from The Hospital for Sick Children (SickKids) have uncovered new genes and genetic changes associated with autism spectrum disorder (ASD) in the largest autism whole genome sequencing analysis to date, providing better understanding into the 'genomic architecture' that underlies this disorder.

Genome 75
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New study uses genetics to identify best sepsis treatment

Drug Discovery World

New research has uncovered how different people respond to sepsis based on their genetics, which could lead to the development of targeted therapies. This provides insight into how an individual’s genetic makeup influences how they respond to sepsis and who would benefit from targeted therapies.

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New approach successfully traces genomic variants back to genetic disorders

Medical Xpress

National Institutes of Health researchers have published an assessment of 13 studies that took a genotype-first approach to patient care. This approach contrasts with the typical phenotype-first approach to clinical research, which starts with clinical findings. The study was published in the American Journal of Human Genetics.

Genome 75
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Gene mapping predicts cancer variants with 100% accuracy

Drug Discovery World

Researchers at the Francis Crick Institute have mapped all the possible outcomes of changes to a tumour-suppressing gene called VHL. It is the first step in a huge research endeavour to unpick the outcomes of tens of thousands of genetic variations in genes associated with cancer.