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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. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.

Treatment 133
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2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Broad Institute

The $400,000 prize, to be shared among the four winners, recognizes their role in creating and advancing a technology that has reshaped how physicians treat leukemia, lymphoma, and multiple myeloma, and is now showing promise in treating autoimmune and infectious diseases. In the early 2000s, both teams began preparing for clinical trials.

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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ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs

Alta Sciences

Nonclinical safety assessment serves as a cornerstone in drug development, providing essential data to identify early signs of potential toxicity and inform subsequent clinical trial design. CAR T cells) WHAT IS THE BENEFIT OF NONCLINICAL CYTOKINE RELEASE EVALUATION? Image Thumbnail_Blog_The Altascientist issue 44_Cytokine Release Assays-3.jpg

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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

Merck’s ENFLONSIA Receives ACIP Endorsement for RSV Prevention in Infants, Marking a Major Milestone in Pediatric Infectious Disease Prevention U.S. The disease burden is disproportionately high among infants under 1 year of age, and even more so among premature infants or those with underlying heart or lung conditions.

Vaccine 52
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The Long Road to End Tuberculosis

Codon

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. 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