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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

Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. Some of these treatments, like K.J.’s, The team that treated K.J. Today, K.J. Today, K.J.

Treatment 133
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mRNA Tech Transfer Phase 2.0 Discussed at G20 Summit

The Pharma Data

2026–2030). Turning growing demand into a sustainable enterprise While there’s a growing market opportunity for mRNA vaccines and therapeutics — ranging from vaccines against seasonal flu and human papillomavirus (HPV) to innovative treatments for cancer — structural barriers remain.

Vaccine 40
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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

HER3 plays a pivotal role in activating downstream signaling pathways through heterodimerization with other receptors like HER2 and can mediate resistance to therapies targeting EGFR and HER2, making it a compelling target for novel treatments in cancers that rely on oncogenic signaling via those related growth factor receptors.

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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. Safety outcomes from the trial further bolster the confidence in MAVYRET’s use.

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Possible adulticidal drugs for Onchocerca volvulus

Drug Target Review

The findings supported the possibility put forward by Cotton et al (2016) concerning the chance of repurposing these drugs for adulticidal treatment. The available treatment for that is minor surgery -to remove the nodules. With accessible and effective treatments, this is feasible. doi: 10.1002/14651858.CD011146.pub2.

Drugs 105