Drug Target Review

JUNE 16, 2025

Multiomics MSI studies provide crucial insights into drug pharmacology and toxicity, supporting the development of innovative therapies including chimeric antigen receptor (CAR) T-cell treatments, oligonucleotide therapeutics, antibody drug conjugates (ADCs) and ribonucleic acid (RNA) therapeutics. Drug Discovery Today 2025, 30 (1), 104275.

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Drug Target Review

DECEMBER 16, 2024

For patients with aggressive diseases, the waiting time for CAR T-cell treatment may be too long. Lentiviral platforms offer the potential of a one-time treatment to generate long-lasting CAR T cells in a patients body, while other RNA-based platforms offer an approach that uses repeat dosing for short-term generation of CAR T cells.

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The Pharma Data

JUNE 9, 2025

Alnylam Secures European Commission Approval for AMVUTTRA® to Treat Cardiomyopathy in ATTR Amyloidosis Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has received European Commission (EC) approval for an expanded indication of its RNAi drug AMVUTTRA® (vutrisiran).

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Treatment Disease RNA Therapies 40

Codon

JUNE 5, 2025

Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.

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DNA RNA Clinical Trials Engineering 83

SugarCone Biotech

JULY 6, 2025

A post-ASCO update on tumor targets DLL3, B7H3 and HER3 July 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and HER3 are compelling tumor antigens to target with cancer therapeutics. However, HER3-DXd missed achieving a statistically significant improvement in overall survival, leading to the withdrawal of its U.S.

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Drug Development Drugs Clinical Trials Therapies 56

BioPharma Drive: Drug Pricing

JUNE 24, 2025

Published June 24, 2025 Gwendolyn Wu Senior reporter post share post print email license Lexeo Therapeutics and two life sciences investors are working together to launch a startup to develop cardiac RNA therapies. Earlier this year, it laid off 15% of its workforce.

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RNA Drugs Therapies Licensing 219

BioPharma Drive: Drug Pricing

JULY 16, 2025

Published July 16, 2025 • Updated 2 hours ago Ben Fidler Senior Editor post share post print email license Sarepta Therapeutics announced a workforce reduction on July 16, 2025. Some Wall Street analysts predicted sales might top $2 billion in 2025. Shares in the company rose in after-hours trading on the news.

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Therapies FDA Clinical Trials Licensing 301

BioPharma Drive: Drug Pricing

JULY 10, 2025

Published July 10, 2025 Delilah Alvarado Staff Reporter post share post print email license The Food and Drug Administration on July 10, 2025, issued Moderna a full approval in children for its COVID-19 vaccine Spikevax. Kennedy Jr., who has pushed for policy changes around mRNA vaccines in particular. Kennedy Jr. Kennedy Jr.

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Vaccine FDA Therapies Licensing 231

Broad Institute

JUNE 25, 2025

By Allessandra DiCorato June 25, 2025 Credit: Xavier and Smillie labs Microscopy image shows intestinal tissue from a Crohn’s patient with fibrosis. Cell network In 2023, Xavier’s lab used single-cell RNA sequencing to create the largest known atlas of single cells from routine biopsies of Crohn’s patients.

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Disease Medical Schools RNA Molecular Biology 111

Broad Institute

MAY 14, 2025

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.

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Engineering DNA RNA Therapies 59

Broad Institute

OCTOBER 16, 2024

William Studier receives the 2024 Merkin Prize in ceremony at the Broad Institute for developing technology used to produce millions of doses of COVID-19 vaccines The groundbreaking, scalable technology is widely used in laboratories around the world today to efficiently produce large amounts of protein and RNA. Merkin (left) and F.

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Vaccine RNA Laboratories Molecular Biology 64

LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). ongoing tirzepatide CVOT to assess head-to-head CV outcomes vs. Trulicity in T2D) will continue to build the case for weight loss (and incretins) in broader metabolic disease and suggest overall healthcare system cost benefits to anti-obesity therapies.

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Small Molecule Trials Therapies Disease 143

Broad Institute

MARCH 27, 2025

Building bridges between Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home Building bridges between Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease is forging connections between Danish and Broad scientists. Here are two of their stories.

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Research DNA Regulations Disease 52

Drug Target Review

JULY 1, 2024

As a non-NSAID, it reduces adverse effects associated with these pain relief drugs, including kidney injury, GI gastritis/ulceration and blood pressure elevation, which can affect patients with cardiovascular disease and the elderly. We set out to improve health by developing a novel non-opioid that avoids potential abuse.

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Treatment Clinical Trials Therapies Molecular Biology 59

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Why are you focusing on liver disease?

Therapies 102

Therapies Disease RNA Engineering 102

Broad Institute

FEBRUARY 11, 2025

Mapping mRNA through its life cycle within a cell By Corie Lok February 11, 2025 Breadcrumb Home Mapping mRNA through its life cycle within a cell Xiao Wangs studies of how and where RNA is translated could lead to the development of better RNA therapeutics and vaccines.

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RNA DNA Science Vaccine 52

Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. The 2025 genetic network paper fit that bill exactly. 2024 Statistical models of organisms have existed for decades.

Engineering 145

Engineering Virus DNA Therapies 145

Codon

MAY 15, 2023

Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. Cell Systems. Cell Systems. From the team at Sherlock Biosciences.

DNA 52

DNA Bioinformatics Engineering Virus 52

Codon

MAY 15, 2023

Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. Cell Systems. Cell Systems. From the team at Sherlock Biosciences.

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DNA Bioinformatics Engineering Virus 52

BioPharma Drive: Drug Pricing

JUNE 30, 2025

Published June 30, 2025 Jonathan Gardner Senior Reporter post share post print email license Moderna announced results from an influenza vaccine clinical trial on June 30, 2025. as early as the 2026-2027 flu season.

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Vaccine Marketing Trials Clinical Trials 100

Broad Institute

MAY 29, 2025

New technique expands cells to sequence DNA and capture fine structural details By Allessandra DiCorato May 29, 2025 Breadcrumb Home New technique expands cells to sequence DNA and capture fine structural details Using this method, scientists discovered key protein and gene activity changes in aging and in a rare disease called progeria.

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DNA RNA Cell Biology Disease 133

Broad Institute

MAY 15, 2025

Evolved gene editor inserts entire genes in human cells By Allessandra DiCorato May 15, 2025 Breadcrumb Home Evolved gene editor inserts entire genes in human cells The new system is the first to use a DNA-mobilizing enzyme called a CRISPR-associated transposase to make targeted gene-sized edits at therapeutically useful levels in human cells.

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DNA Laboratories RNA Disease 133

Broad Institute

JANUARY 27, 2025

A genome-wide atlas of cell morphology reveals gene functions By Allessandra DiCorato January 27, 2025 Breadcrumb Home A genome-wide atlas of cell morphology reveals gene functions PERISCOPE, a technique for genome-wide imaging screens, is helping Broad scientists understand the connections between genes and traits. Online January 27, 2025.

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DNA RNA Science Research 131

Elrig

MARCH 10, 2025

Research & Innovation 2025 Keynote Speakers Announced Cambridge, UK, 24 January 2025: The European Laboratory Research & Innovation Group (ELRIG), a not-for-profit, volunteer-led organisation for the drug discovery community, today announced the keynote speakers for Research & Innovation 2025.

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Drug Target Review

APRIL 1, 2025

Advances in biotechnology are driving significant progress in the treatment of rare diseases, making it possible to develop targeted therapies for previously untreatable conditions. Our vision for 2025 is to get into the clinic with our candidate drug for tackling H-ABC. It also received two grants from Innovate UK, totalling 2.5

Disease 52

Disease Therapies Clinical Trials Cell Biology 52

Broad Institute

MARCH 27, 2025

A Novo Nordisk - Broad collaboration is connecting Denmark-based and Broad researchers By Corie Lok March 27, 2025 Breadcrumb Home A Novo Nordisk - Broad collaboration is connecting Denmark-based and Broad researchers The Novo Nordisk Foundation Center for Genomic Mechanisms of Disease has built a bridge between Danish and Broad scientists.

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Research DNA Regulations Disease 75

keypoint

JANUARY 8, 2025

Our first Fellows Spotlight of 2025 goes to Keystone Symposia Fellow Dr. Abigael Cheruiyot! Studying RNA surveillance mechanisms, they identified nonsense mediated decay as a potential target for novel cancer therapies. She is very passionate about both science and promoting equity within the research community.

Medical Schools 59

Medical Schools RNA Laboratories Doctors 59

Codon

MAY 8, 2025

On biology At some point, your high-school biology teacher probably told you (or will tell you) this story about how life works: First, your DNA gets transcribed into matching RNA. Next, that RNA gets translated into protein. Then the protein does Protein Stuff. The pre-mRNA is now all grown up and has graduated to being regular mRNA.

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DNA RNA Molecular Biology Regulations 79

Codon

FEBRUARY 19, 2025

Researchers today often spend months trying to figure out whether a genetic mutation causes disease, simply because laboratory experiments are slow. ESM is a protein language model that captures certain aspects of protein structure and function, for example, but is blind to DNA or RNA-level features that drive cell behavior.

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DNA Laboratories Engineering RNA 109

Drug Target Review

NOVEMBER 27, 2024

For example, the European Medicines Agency (EMA), in its Regulatory Science Strategy to 2025 , has highlighted the critical role of biomarker discovery, qualification and utilisation in accelerating precision medicine. Finally, its application in urine samples presents additional challenges compared to serum.

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Drug Development Regulations Laboratories FDA 52

Drug Target Review

MAY 8, 2025

Huntington’s disease (HD) is a devastating neurodegenerative disorder with profound generational impacts. This hereditary pattern makes HD particularly challenging from both medical and societal perspectives: a condition with a known genetic cause but, to date, no disease-modifying treatments.

Disease 111

Disease Treatment DNA Therapies 111

Broad Institute

JANUARY 7, 2025

A clinical genomicist harnesses team-based science to help rare-disease patients By Allessandra DiCorato January 7, 2025 Breadcrumb Home A clinical genomicist harnesses team-based science to help rare-disease patients Heidi Rehm convinced labs and scientists to work together and share data.

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Disease Science Laboratories Medical Schools 121

Broad Institute

JANUARY 16, 2025

By Allessandra DiCorato January 16, 2025 Credit: Scott Sassone, Broad Communications Tara McDonald (left) and Steve McCarroll (right) are coauthors of a new paper describing a biological mechanism underlying Huntington's disease. The cumulative death of many such cells leads to the symptoms of Huntingtons disease.

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Disease DNA Medical Schools RNA 130

Broad Institute

MAY 20, 2025

Scientists build cell line atlas of deadly cancers of the bile ducts and gallbladder By Leah Eisenstadt May 20, 2025 Breadcrumb Home Scientists build cell line atlas of deadly cancers of the bile ducts and gallbladder Researchers reveal new subtypes and potential therapeutic targets for biliary tract cancers, which lack effective treatments.

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Medical Schools Treatment Bioinformatics Hospitals 133

Codon

JANUARY 26, 2025

Astrologers of the time believed that the disease’s periodic return was influenced by the location of the stars. By contrast, smallpox and measles vaccines are more than 95 percent effective at preventing disease, a level sufficient to have eradicated smallpox entirely. Formaldehyde had precedent in medicine.

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Vaccine Virus Immune Response Clinical Trials 116

Codon

MAY 29, 2025

This deadly structure allows it to kill its victims by preventing their cells from making messenger RNA (mRNA) through inhibition of the enzyme RNA Polymerase II. The ADC approach allows healthy cells to avoid the deadly effects suffered by the cancer cells as their RNA polymerase II enzymes are shut down.

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Drugs DNA RNA Laboratories 109

Codon

MAY 5, 2025

This allowed for the isolation, purification, and study of minute amounts of DNA, RNA, proteins, and cells — tasks ill-suited to the larger centrifuges. Doctors diagnose diseases and separate blood into components for testing and treatment. ” Asimov Press (2025). Cite: McLain, R. “Making the Centrifuge.”

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