Drug Target Review

JUNE 16, 2025

1 Continuous advancements in sensitivity and spatial resolution support the role of MSI in pharmaceutical research. By integrating MSI with histopathology, clinical chemistry and DMPK data, researchers can develop mechanistic hypotheses of toxicity, improving decision making and reducing unnecessary follow-up studies. References [1].

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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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Fierce BioTech

MARCH 14, 2025

Advancing the Future of Biomarker-Driven Immunotherapy swheeler Fri, 03/14/2025 - 13:43 Mon, 04/21/2025 - 12:00 Resource Type Webinar Eduard Porta-Pardo Can spatial biomarkers revolutionize patient stratification for immune checkpoint inhibitors? On Demand Start Date Mon, 04/21/2025 - 13:00 End Date/Time Mon, 04/21/2025 - 13:00

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RNA Clinical Trials Therapies Trials 59

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

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. The statement did not specify where the non-viral delivery technology the new startup will use comes from. “We

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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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Drug Target Review

DECEMBER 16, 2024

Today, researchers are exploring new ways to simplify and deliver CAR T cells more quickly, or even generate them directly in the patients body, an area of research led by Umoja Biopharma. Umoja aims to dose its first patients near the end of 2024 with clinical updates expected in 2025.

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LifeSciVC

JANUARY 16, 2024

While names like DNLI, JNJ, DYN, and RNA were real pioneers in the space for delivery of antibodies and oligos, more and more companies are developing their own TfR1 shuttles or partnering to bring in the capability. TfR1 is almost becoming table stakes for Pharma and biotech looking to deliver to the brain and / or muscle.

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Drug Target Review

JULY 1, 2024

Using the 10X Genomics Chromium platform, we conducted ribonucleic acid (RNA) sequencing on the midbrain’s PAG region in animals treated with SRP-001, ApAP and a vehicle control, focusing on gene expression changes related to pain processing. He was bestowed the 2024 NIH HEAL Director’s Trailblazer Award for this work.

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Treatment Clinical Trials Therapies Molecular Biology 59

Codon

JUNE 5, 2025

The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. This article provides a summary of the major CRISPR systems, 2 including the naturally occurring CRISPR-Cas9, -Cas12, and -Cas13 systems, as well as base editors, prime editors, and the recently uncovered bridge RNA system.

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Broad Institute

FEBRUARY 26, 2025

Why brain cancer is often resistant to immunotherapy By Allessandra DiCorato February 26, 2025 Breadcrumb Home Why brain cancer is often resistant to immunotherapy Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance.

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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. Lingjia Kong, a research scientist in the Xavier lab, is the first author on the study. Currently, the only treatment option for these gut “strictures” is surgery.

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Codon

APRIL 14, 2025

While I deeply respect that tolerance, it can keep the field stuck: if we see pain as par for the course, it’s easy to overlook chances to make biological research faster, cheaper, and more efficient. Of course, biological research is shaped by different pressures than hardware projects.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. When combined with our in-house GMP-level RNA chemistry lab, Ochre Bio is able to progress from a new target to generating human data on a lead therapy in days. What does Ochre Bio do, can you give me an overview of your work?

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

Broad Institute

MAY 14, 2024

William Studier for development of widely used protein- and RNA-production platform By Corie Lok May 14, 2024 Breadcrumb Home Merkin Prize in Biomedical Technology awarded to F. Merkin Prize in Biomedical Technology for his development of an efficient, scalable method of producing RNA and proteins in the laboratory.

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Codon

SEPTEMBER 29, 2024

But, regardless of which was first, they all operated with the same core data as their mechanism for understanding life: messenger RNA ( mRNA ). The 2025 genetic network paper fit that bill exactly. The lab created a plan with eight research institutions across three continents. As such, they were quietly abandoned.

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Codon

MAY 15, 2023

🔥 Week in Review Biotech research highlights in under 5 minutes. For one of the studies , researchers collected blastocysts from cynomolgus monkeys, the long-tailed macaques native to southeast Asia. For another study , researchers made mouse models with prime editors encoded in the germline of various tissues.

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DNA Bioinformatics Engineering Virus 52

Codon

MAY 15, 2023

🔥 Week in Review Biotech research highlights in under 5 minutes. For one of the studies , researchers collected blastocysts from cynomolgus monkeys, the long-tailed macaques native to southeast Asia. For another study , researchers made mouse models with prime editors encoded in the germline of various tissues.

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DNA Bioinformatics Engineering Virus 52

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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Research Laboratories Medical Schools Engineering 52

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

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

APRIL 3, 2025

Scientists have developed a way to scale up spatial genomics and lower costs By Allessandra DiCorato April 3, 2025 Breadcrumb Home Scientists have developed a way to scale up spatial genomics and lower costs A new computational approach eliminates time-intensive imaging, enabling high-resolution spatial mapping of gene expression in tissue.

DNA 119

DNA RNA Molecular Biology Laboratories 119

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.

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DNA RNA Science Research 131

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

Codon

APRIL 10, 2025

It is the subject of more than 100,000 published research papers. The other 30 percent — the so-called “dry mass” — is composed of everything else: DNA, RNA, proteins, lipids, and ions. About 20 percent of the dry mass is RNA, 10 percent is lipids, and the rest is ions and signaling molecules.

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DNA RNA Engineering Research 93

Codon

FEBRUARY 16, 2025

They are depicted in research papers as dense tangles of arrows, triangles, and other symbols; similar to the diagrams found in electronic engineering textbooks. The results convinced skeptics that researchers could engineer predictable circuits into living cells. 2 Many modern gene circuits, though, are incredibly complicated.

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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.

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Medical Schools RNA Doctors Laboratories 59

Codon

FEBRUARY 19, 2025

Today, Arc Institute (a research nonprofit in Palo Alto, California) and NVIDIA launched a broader AI model for biology, called Evo 2 , that can do much the same for entire genomes. Researchers today often spend months trying to figure out whether a genetic mutation causes disease, simply because laboratory experiments are slow.

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DNA Laboratories Engineering RNA 105

Codon

MAY 8, 2025

I expect this number to rise a bit over time, as the newest methods use deep learning and research is ongoing.) 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.

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Drug Target Review

APRIL 1, 2025

Co-founder and CEO Dr Dan Williams brings over 20 years of experience in biochemistry and drug development, with a strong background in research and clinical trials. In this interview, Williams shares the companys plans for 2025, the challenges of treating rare diseases, and how gene-silencing technology could unlock new treatment options.

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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. LC-MS/MS also surpasses ELISA in sensitivity, making it a useful technique for detecting low-abundance species. per sample.

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Drug Target Review

MAY 8, 2025

This dilemma has driven our research at Life Edit towards allele-selective approaches that target only the toxic mutant huntingtin gene while preserving the healthy copy a precision medicine approach that addresses the disease mechanism while minimising potential adverse effects. Accessed March 2025. Accessed March 2025.

Disease 111

Disease Treatment DNA Therapies 111

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 researchers found that DNA tracts with 40 or more CAG repeats grow until they are hundreds of repeats long.

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Disease DNA Medical Schools RNA 130

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. Thats what makes her so convincing.

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Disease Science Laboratories Medical Schools 121

Broad Institute

FEBRUARY 5, 2025

Mutations in two gene pairs point to a promising drug target in 5 percent of adult cancers By Allessandra DiCorato February 5, 2025 Breadcrumb Home Mutations in two gene pairs point to a promising drug target in 5 percent of adult cancers Targeting the PELO gene could be a way to kill tumor cells in cancers with one of two common genetic mutations.

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Protein Production DNA Drugs RNA 100

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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