Archives September 2019

ICER’s Leader: How to Assess the Value of Cell and Gene Therapies

Science is producing a growing number of gene and cell therapies to treat spinal muscular atrophy, leukemia and other conditions, but payers and providers don’t have the information they need to determine what they should pay for these treatments. That’s according to Steven Pearson, MD, founder and president of Boston-based Institute for Clinical and Economic

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Pentagon’s research agency wants to use gene editing to protect troops against chemical and biological weapons

Instead of augmenting super-soldiers, DARPA wants to boost troops’ natural defenses against engineered diseases — and even undo gene-editing altogether. The Pentagon’s research agency wants to explore the possibility of editing a soldier’s genetic makeup to protect against chemical and biological attacks. Defense Advanced Research Projects Agency director Steven Walker said Monday that he believes

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Novartis in political row after denying Belgian toddler compassionate access to gene therapy

The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds. According to The Brussels Times, the family of the toddler named Pia, who suffers from the ultra-rare disease spinal muscular atrophy (SMA) have been running a crowdfunding campaign

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FDA fast tracks novel treatment for Guillain-Barré syndrome

Annexon Biosciences, a clinical-stage biopharmaceutical company developing a pipeline of novel therapies for patients with classical complement-mediated disorders of the body, eye and brain, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ANX005 for the treatment of Guillain-Barré Syndrome (GBS), a rare, acute, antibody-mediated autoimmune disease impacting

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EU-LIFE open letter urges EU Council & EU Parliament to prioritise R&I by committing an appropriate budget for Horizon Europe

EU-LIFE, the alliance of 13 leading Life Science Research Institutes in Europe urges the EU Council and the European Parliament to double Horizon Europe’s budget compared to Horizon 2020 (H2020), to 150 Billion Euros and within it, to double the budget for discovery research, including the well-proven European Research Council. In addition, we strongly support the

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Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning

Source https://www.nature.com/articles/s41467-019-12281-8#Sec14 Highly specific Cas9 nucleases derived from SpCas9 are valuable tools for genome editing, but their wide applications are hampered by a lack of knowledge governing guide RNA (gRNA) activity. Here, we perform a genome-scale screen to measure gRNA activity for two highly specific SpCas9 variants (eSpCas9(1.1) and SpCas9-HF1) and wild-type SpCas9 (WT-SpCas9) in

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University of Akron: Thesis Student 3D Prints PPF Structures for Bone Regeneration Applications

In ‘Synthesis, characterization and 3D printing of linear and star-shaped poly(propylene fumarate) for medical applications,’ Yuanyuan Luo offers a PhD dissertation for The University of Akron on the subject of additive manufacturing and new polymer synthetics for use in the medical realm. As materials science continues to expand, researchers are drawn to exploring the uses of 3D

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Researchers from University of Regina develop a promising new ‘Trojan horse’ treatment for multiple sclerosis

We often take our immune and nervous systems for granted. We assume that our immune system will protect us from diseases and when pathogens invade our body. Likewise, we assume that our nervous system will take information from the environment, relay it to our brain and then allow our brain to move muscles. But what

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