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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

Muldoon, a baby boy born without the ability to process dietary protein properly, had become the first person to be treated with a customized gene editing therapy. wasn’t the first patient to receive base editing therapy. He needed a personalized, one-of-a-kind therapy. Today, K.J. The result appears to be a major success.

Treatment 133
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The Long Road to End Tuberculosis

Codon

But they will need to do so if they want to meet the goals set out by the End TB Strategy , namely, to reduce TB cases by 80 percent and curb deaths by 90 percent before 2030. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent. Subscribe to Asimov Press.

Vaccine 114
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Targeting HER3 – a little wave of drug development that’s about to get a lot bigger

SugarCone Biotech

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. These results are notable given the poor response of NRG1 fusion driven cancers to chemo- and immuno-therapies. These activities are pro-oncogenic (reviewed here: [link] ).

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Best-in-Class Solutions Accelerate Development of GLP-1 Therapeutics

PPD

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. The SELECT trial set out to understand whether the drug has similar effect on patients without diabetes.

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FDA Approves Expanded Use of AbbVie’s MAVYRET for Acute Hepatitis C

The Pharma Data

Food and Drug Administration (FDA) approved a label expansion for MAVYRET® (glecaprevir/pibrentasvir), the company’s oral, pangenotypic direct-acting antiviral (DAA) therapy. If treated early with safe and effective therapies, providers can cure virtually all patients with hepatitis C before it escalates. Global and U.S.

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Sentiment & Themes Emerging From JPM 2024

LifeSciVC

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Additional trials (e.g.,

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The Race for Ozempic Alternatives: Unlocking the Future of Weight Loss Medication

DrugBank

adults using obesity medications by 2030, further underlining the growing significance of this sector. On the other hand, Amgen revealed early-stage trial data on a promising once-a-month, weight loss medication, signaling its entry into this competitive arena. Goldman Sachs analysts anticipate a significant rise in the number of U.S.