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Researchers reverse the in vitro and in vivo effects of the mutation that causes Stormorken syndrome

Medical Xpress

That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material.

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Vertex secures FDA clearance for infants’ cystic fibrosis therapy

Pharmaceutical Technology

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results. The medicine is currently available for use in more than 30 countries.

In-Vitro 246
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BMS’s repotrectinib to have FoundationOne genomic sequencing companion diagnostic

Pharmaceutical Technology

Foundation Medicine has announced it will supply a tissue-based test as a companion diagnostic for Bristol Myers Squibb’s (BMS) recently acquired ROS1/TRK inhibitor repotrectinib. FoundationOne CDx is a sequencing-based in vitro diagnostic device that can identify alterations in 324 genes from tumour samples.

Genome 130
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Researchers revive abandoned technique in effort to make artificial human eggs in a test tube

STAT News

Using a common cloning technique, researchers removed the genetic material from one female’s eggs and replaced them with nuclear DNA from the skin cells of another. Then with a novel chemical cocktail, they nudged the eggs to lose half their new sets of chromosomes and fertilized them with mouse sperm. Read the rest…

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Evotec and Variant Bio partner to develop fibrosis treatments

Drug Discovery World

Evotec will identify best-in-class small molecules targeting a key fibrotic pathway with strong genetic support identified by Variant Bio and progress the programme towards the selection of a clinical development candidate(s) using its R&D platform.

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14th Annual RNA Therapeutics Conference

pharmaphorum

Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Heads of Translational Medicine.

RNA 52
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Stem cells’ versatility in drug development  

Drug Discovery World

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process. This article is sponsored by Charles River.