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RNA interference method could treat muscular dystrophy

Drug Discovery World

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . The genetic anomaly blocks the chemical glycosylation of a biologically important protein. Modifying RNA function. Image credit: Mariko Taniguchi-Ikeda from Fujita Health University

RNA 52
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Rome Therapeutics raises $77m for junk DNA-targeting drugs

pharmaphorum

Startup Rome Therapeutics has raised $77 million in second-round financing to help mine sequences of DNA – which were dismissed for years as ‘junk’ – for hidden treasure. Most drug discovery programmes target the roughly 2% of the human genome which encodes for protein.

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Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder

XTalks

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

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Conversations from ESMO Targeted Anticancer Therapies Congress 2024 

Drug Discovery World

More importantly, it also highlighted that significant challenges remain in selecting the right therapies for the right patients despite the progress made through genomic testing and implementation of circulating tumour (ct) DNA analyses. Results of that Phase I trial are expected to be presented later this year.

RNA 64
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HIV vaccines suffer setbacks but new approaches could turn the tide

Pharmaceutical Technology

Only a few weeks into the new year, the prospect of getting a successful advanced HIV vaccine shrank after the discontinuation of yet another late-stage trial. This marks the second time one of Janssen’s HIV vaccines failed after another showed disappointing results in the Phase IIb Imbokodo trial in August 2021.

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Gene Therapy and Pharmacokinetics

Camargo

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. For example, it is beneficial to administer RNA treatments for some lung diseases directly to the disease site through inhalation.

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The Utility of Liquid Biopsy in Oncology Clinical Trials

XTalks

Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management.