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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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2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Broad Institute

The work of June, Levine, Rivire, and Sadelain laid the foundation for a whole new class of treatments. In the early 2000s, both teams began preparing for clinical trials. Their contributions have saved lives and opened doors for future innovations that will continue to transform human health.

Therapies 119
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Navigating Regulatory Hurdles in Drug Development

DrugBank

The failure rate in clinical trials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance. Even drugs that complete clinical trials may face delays or rejections if submission documents are incomplete or do not align with regulatory expectations.

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Takeda to seek approval of new kind of narcolepsy drug after study data

BioPharma Drive: Drug Pricing

According to Takeda, oveporexton hit every main and secondary trial endpoint, helping improve patient-reported scores on measures of wakefulness, excessive daytime sleepiness, muscle weakness, and other symptoms after 12 weeks of treatment. You can unsubscribe at anytime. Takeda will file an approval application in the U.S.

Drugs 187
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mRNA Tech Transfer Phase 2.0 Discussed at G20 Summit

The Pharma Data

This meant putting the fundamentals of mRNA production directly into the hands of local innovators — a dramatic departure from previous models that kept much of vaccine production within a small number of firms in high-income settings. mRNA Technology Transfer Programme Moves Into Phase 2.0, 2026–2030).

Vaccine 40
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Merck’s ENFLONSIA Receives ACIP Recommendation for Protecting Infants from Severe RSV

The Pharma Data

CDC panel backs long-acting monoclonal antibody to protect infants during their first RSV season; drug also included in federal Vaccines for Children program Merck , operating as MSD outside the United States and Canada, has announced a key regulatory milestone for its respiratory syncytial virus (RSV) prevention program. What Is ENFLONSIA™?

Vaccine 52
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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. Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB.

Vaccine 115