Fierce BioTech

APRIL 10, 2025

On Demand Start Date Wed, 06/18/2025 - 13:00 End Date/Time Wed, 06/18/2025 - 13:00

Clinical Trials 52

Clinical Trials RNA Trials Therapies 52

Alta Sciences

FEBRUARY 19, 2025

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.

Drug Development 52

Drug Development Clinical Pharmacology Immune Response Drugs 52

Broad Institute

FEBRUARY 24, 2025

Although this ancestral replicase appears to have been lost, key aspects of RNA-catalyzed RNA replication can be studied by proxy with the use of modern RNA enzymes (ribozymes) generated by in vitro evolution. However, none of these RPRs was capable of self-replication.

RNA 52

RNA Molecular Biology Laboratories Engineering 52

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.

Engineering 52

Engineering DNA RNA Therapies 52

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

RNA 59

RNA Clinical Trials Therapies Trials 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.

Vaccine 64

Vaccine RNA Laboratories Molecular Biology 64

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.

Small Molecule 143

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.

Research 52

Research DNA Regulations Disease 52

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.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

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. Online February 26, 2025.

Immune Response 132

Immune Response Treatment RNA Clinical Trials 132

Codon

APRIL 14, 2025

Some, like predicting microbial growth rate from genetics , predicting CRISPR editing efficiency at a locus given a guide RNA , and protein stability prediction are in progress but would benefit from more support, especially to make solutions widely applicable and available to potential users. ” Asimov Press (2025).

Protein Expression 92

Protein Expression Engineering Research RNA 92

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.

RNA 52

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.

RNA 84

RNA Production Molecular Biology Laboratories 84

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. There was scFormer in 2022, scGPT in 2023, and plenty of others. As such, they were quietly abandoned.

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.

DNA 52

DNA Bioinformatics Engineering Virus 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.

DNA 119

DNA Laboratories RNA Disease 119

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

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.

Research 52

Research Laboratories Medical Schools Engineering 52

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.

DNA 131

DNA RNA Science Research 131

Codon

FEBRUARY 16, 2025

Building a Circuit Much like electronic circuits, which use wires and transistors to create or process signals, a synthetic gene circuit is a network of biological parts — DNA, RNA, and proteins — that process inputs and generate outputs. Also, RNA polymerase moves in bursts along DNA, rather than at a steady pace.

DNA 96

DNA RNA Engineering Small Molecule 96

Codon

APRIL 10, 2025

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. Similar considerations apply for DNA, RNA, and all the other molecules, too.

DNA 96

DNA RNA Engineering Regulations 96

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.

DNA 71

DNA RNA Molecular Biology Regulations 71

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.

Research 75

Research DNA Regulations Disease 75

Drug Target Review

APRIL 1, 2025

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. Vision for 2025: A major step towards clinical trials Since its founding, SynaptixBio has made significant progress in advancing its research and development efforts.

Disease 52

Disease Therapies Clinical Trials Cell Biology 52

Codon

FEBRUARY 19, 2025

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. Evo 2 was trained on DNA sequences but can make predictions about myriad aspects of biology, including RNA stability and protein structures. & McCarty N.

DNA 107

DNA Laboratories Engineering RNA 107

Drug Target Review

DECEMBER 16, 2024

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. Umoja aims to dose its first patients near the end of 2024 with clinical updates expected in 2025.

Therapies 52

Therapies Clinical Trials Treatment Trials 52

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.

Drug Development 52

Drug Development Regulations Laboratories FDA 52

Drug Target Review

MAY 8, 2025

We observed robust AAV vector distribution across key brain regions along with significant guide RNA and nuclease expression. Accessed March 2025. Accessed March 2025. ” Presented at the 20th Annual Huntington’s Disease Therapeutics Conference, February 24-27, 2025, Palm Springs, CA. . ” HDSA.org.

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. Online January 16, 2025. Funding This work was supported by CHDI Foundation, Inc., DOI: 10.1016/j.cell.2024.11.038.

Disease 130

Disease DNA Medical Schools RNA 130

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.

Protein Production 100

Protein Production DNA Drugs RNA 100

Codon

MAY 15, 2025

Others express non-coding RNAs, including amino acid-carrying transfer RNAs pivotal to protein synthesis, or microRNAs that trigger the breakdown of messenger RNA and inhibit gene expression. ” Asimov Press (2025). These mutations may partly account for the reproducibility crisis. Sign up for Asimov Press.

DNA 78

DNA Laboratories RNA Regulations 78

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.

Disease 121

Disease Science Laboratories Medical Schools 121

SugarCone Biotech

APRIL 8, 2025

The most typical genetic cargo for LNP-mediated in vivo CAR T cell production is messenger RNA (mRNA). Also notable is that financing support has been robust through 2024 (Interius) and into 2025 (Umoja). Whether the shrill anti-RNA stance taken by the new head of FDA will impact RNA-based therapeutics is unclear.

Therapies 57

Therapies RNA Packaging Virus 57

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. Pharmaceutical and biotech researchers produce vaccines, extract DNA and RNA, separate nanoparticles, and prepare samples. ” Asimov Press (2025). Cite: McLain, R.

Engineering 57

Engineering DNA Science Laboratories 57