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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. The package insert for the drug, Elevidys , warns of adverse effects of acute liver injury and inflammation around the heart and muscles. million DNA bases.

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Article FDA Thank You FDA’s oncology advisory committee to consider MRD as a multiple myeloma endpoint

Agency IQ

Between 2012 and 2018, the five-year relative survival rate for patients with MM was 57%. The ideal scenario is to have a clinical trial that prospectively evaluates these endpoints, he said, providing a data package the FDA can use to evaluate such alternative endpoints.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g., CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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Beyond Steel Tanks

Codon

In the case of semaglutide, those cells are Saccharomyces cerevisiae— also known as Baker’s yeast — engineered to secrete a peptide precursor that is later purified, chemically modified, packaged into an injectable or tablet form, and then shipped around the world. Continuing this method may not scale.

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Medicines Patent Pool signs sublicences with Aurobindo, Cipla and Viatris to produce generic versions of ViiV Healthcare’s innovative long-acting HIV prevention medicine

The Pharma Data

Unitaid is working with implementing partners to ensure optimal use as part of a PrEP package that enables choice, and with the Coalition and other international partners to make long-acting PrEP options equitably accessible as quickly as possible.” Shionogi became a ViiV shareholder in October 2012.

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Analysis Chemical Thank You What we expect the EPA to do in April 2024

Agency IQ

The EPA is also proposing our response to a petition regarding the continued validity of EPA’s 2012 determination (and September 2017 affirmation) that participation in the Cross State Air Pollution Rule (CSAPR) meets the Regional Haze Rule’s criteria for an alternative to the application of source-specific BART.

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Codon Digest: Hackathon Prize Winners

Codon

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. Anyone who has tried to engineer a cell knows how tedious it can be.

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