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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. Some of these treatments, like K.J.’s, They plan to harness scientific, medical, regulatory, and manufacturing innovations to enable on-demand genetic treatments like K.J.’s

Treatment 133
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The Long Road to End Tuberculosis

Codon

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. 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.

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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Gilead Advances in HIV Market With Twice-Yearly Yeztugo, Leaving GSK Behind

The Pharma Data

Gilead’s HIV Business: Already a Powerhouse Gilead has long been a dominant player in the HIV treatment and prevention market. billion—coming from Biktarvy, a once-daily triple-combination therapy for people living with HIV. Descovy, another key product used for both treatment and PrEP, added $2.1

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Pharma Contract Sales Outlook 2025–2030: Multilingual Teams Drive Global Expansion

The Pharma Data

Billion by 2030: Strategic Insights, Regional Trends, and Technological Shifts Reshaping the Landscape The global pharmaceutical contract sales outsourcing (CSO) market is entering a transformative phase, with its value expected to grow from $17.3 billion by 2030 , reflecting a compound annual growth rate (CAGR) of 6.2%.

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

PPD

Initially approved for treatment of Type 2 diabetes and later for obesity studies in GLP-1 are now expanding in other indications such as obstructive sleep apnea (OSA), heart failure with preserved ejection fraction (HFpEF) and chronic kidney disease (CKD).

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

The Pharma Data

FDA Expands Approval of AbbVie’s MAVYRET® as First and Only 8-Week Treatment for Acute Hepatitis C in Adults and Children Aged 3 and Above AbbVie has received a significant regulatory boost for its hepatitis C treatment portfolio as the U.S. However, progress toward that target has been slow. Global and U.S.