2024 and RNA - Drug Discovery Digest

NGS is evolving: collaboration and tech lead the way

Drug Target Review

JULY 15, 2025

Their latest assay processes four to 24 libraries per batch and detects gene amplifications, fusions, deletions and other cancer biomarkers from both DNA and RNA inputs. Their latest assay processes four to 24 libraries per batch and detects gene amplifications, fusions, deletions and other cancer biomarkers from both DNA and RNA inputs.

RNA

RNA DNA Laboratories Science

Researchers map communities of single cells in metastatic breast cancers

Broad Institute

OCTOBER 30, 2024

Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.

Research

Research RNA Medical Schools Treatment

The biotech mapping thousands of hidden therapeutic clues

Drug Target Review

JUNE 30, 2025

This allows the team to spot previously unannotated proteins and study them across multiple biological dimensions – from structure to function, from RNA expression to genetic association. . “RiboSeq technology gives us a dynamic, real-time snapshot of all of the proteins that are being translated in a cell,” Lepore explains.

Disease

Disease Medical Schools Science Therapies

New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

JUNE 9, 2025

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases.

Disease

Disease Treatment Engineering Protein Production

A spatial approach to understanding drug dynamics using mass spectrometry imaging

Drug Target Review

JUNE 16, 2025

2024) 6 demonstrated MSI’s ability to distinguish biliary toxicants by revealing distinct PKs within hepatocytes and bile duct cells, informing the development of safer alternatives. 2024, 52 (11), 1181–1186. npj Imaging 2024, 2 , 20. 2024, Article ASAP. 4 Similarly, Groseclose et al. Furthermore, Yang et al.

Drugs

Drugs Organic Chemistry Laboratories Pharmaceuticals

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development

Alta Sciences

FEBRUARY 19, 2025

Regulatory Considerations for Oligonucleotide Drug Development and Safety In 2024, the U.S. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy. Our experts stay up to date on all new and evolving regulations to guarantee regulatory compliance in your studies.

Drug Development

Drug Development Clinical Pharmacology Immune Response Drugs

Sanofi Showcases Rare Disease Pipeline Innovations at ISTH

The Pharma Data

JUNE 6, 2025

and EU, in late 2024 as the first therapy of its kind. Qfitlia was approved in multiple regions, including the U.S. By lowering AT—a natural anticoagulant—Qfitlia shifts the coagulation balance toward thrombin generation, thereby enhancing clot formation in individuals with clotting factor deficiencies.

Disease

Disease Therapies Treatment International

A Visual Guide to Genome Editors

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.

DNA

DNA RNA Clinical Trials Engineering

The Long Road to End Tuberculosis

Codon

NOVEMBER 3, 2024

These targeted different bacterial components, including RNA polymerases that control gene expression plus enzymes involved in synthesizing the bacterium’s unique cell wall. ” Asimov Press (2024). The researchers were left with 40 drug candidates. Cite: Nahas, Kamal. “The Long Road to End Tuberculosis.”

Vaccine

Vaccine Trials Therapies Treatment

In vivo CAR T: Faster, cheaper, and more effective cancer care

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

Therapies Clinical Trials Treatment Trials

Targeting HER3 – a little wave of drug development that’s about to get a lot bigger

SugarCone Biotech

JULY 6, 2025

This drug received accelerated approval in December 2024 for pancreatic cancer and non-small-cell lung cancers (NSCLC) carrying NRG1 gene fusions, only 11 years after the first NRG1 gene fusion paper was published ( [link] ). The approval was based on a small Phase 1/2 study run by the MSKCC and collaborators.

Drug Development

Drug Development Drugs Clinical Trials Therapies

Lexeo to help launch spinout around RNA drugs for the heart

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 Can they keep it?

RNA

RNA Drugs Therapies Licensing

Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

BioPharma Drive: Drug Pricing

JULY 16, 2025

Sarepta became profitable in 2024, and believes it can stay in the black even if Elevidys only draws revenue from ambulatory patients, executives said on that June call. 4, 2024 Adeline Kon/BioPharma Dive Deep Dive // Emerging biotech Biotech IPOs are the industry’s lifeblood. By Jonathan Gardner • Sept. Track how they’re performing.

Therapies

Therapies FDA Clinical Trials Licensing

Levers for Biological Progress

Codon

NOVEMBER 1, 2024

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. SeqFISH, developed by Long Cai’s group at Caltech, maps the spatial positions of thousands of RNA, DNA, or protein molecules within single cells.

DNA

DNA RNA Molecular Biology Laboratories

Atlas XIV And Reflections On The VC Math Problem

LifeSciVC

DECEMBER 5, 2024

combinatorial chemistry, human genetics, functional genomics, mRNA, I/O, CRISPR, RNA editing, and many others). Over the past decade (2015-2024), looking only at meaningful exits above $100M values: M&As of private biotechs have had a median and average of $270M and $600M, respectively. Top decile is $3B or so.

Marketing

Marketing RNA Drugs Science

Optical density and absorbance measurements | BMG LABTECH

BMG Labtech

DECEMBER 20, 2024

Absorbance measurements at this specific wavelength can be used to determine DNA or RNA concentrations quickly. Source: tip 3 in OD600 measurement: 4 facts and tips to know for bacterial culture growth [link] Accessed October 31 2024. Nucleic acids show natural absorbance at 260 nm (Fig. Online corrected version: (2008) "Absorbance".

DNA

DNA Science Laboratories RNA

FDA’s Issues Draft Guidance on Accelerated Approval: A Substantial Evidentiary and Procedural Overhaul to this High-Profile Pathway

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

Sasinowski On December 5, 2024, FDA published a new draft guidance on accelerated approval providing a much needed and substantial update to its guidance on the pathway. FDAs December 5, 2024 draft guidance on accelerated approvals appears intended to cover each of these topics even if not by dividing them precisely along those lines.

FDA

FDA Clinical Trials Trials Therapies

A covalent compound selectively inhibits RNA demethylase ALKBH5 rather than FTO

Covalent Modifiers

FEBRUARY 25, 2024

2024 , DOI: 10.1039/D3CB00230F N6-Methyladenosine (m6A) is the most prevalent mRNA modification and is required for gene regulation in eukaryotes. TD19 irreversibly modifies the residues C100 and C267, preventing ALKBH5 from binding to m6A-containing RNA.

RNA

RNA Cell Based Assays Regulations Research

AZVUDINE

New Drug Approvals

JULY 18, 2025

2024, 67, 4376−4418 Azvudine (1). It acts as an RNA-dependent RNA polymerase (RdRp) inhibitor, the same mechanism as the previously approved molnupiravir and remdesivir. It acts as an RNA-dependent RNA polymerase (RdRp) inhibitor, the same mechanism as the previously approved molnupiravir and remdesivir.

RNA

RNA Clinical Trials Trials Virus

Blood Test Spots Early Pancreatic Cancers With 97% Accuracy

Drugs.com

APRIL 8, 2024

MONDAY, April 8, 2024 -- A blood test appears capable of detecting early-stage pancreatic cancers with up to 97% accuracy, a new study reports.The test looks for eight small RNA particles and eight larger DNA markers shed by pancreatic cancers,

RNA

RNA DNA

In Early Research, Heart Drugs Show Promise in Fighting Muscular Dystrophy

Drugs.com

JANUARY 3, 2024

3, 2024 -- A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests.Myotonic dystrophy type 1 (DM1) is caused by abnormal RNA. WEDNESDAY, Jan.

RNA

RNA Research Drugs

Sentiment & Themes Emerging From JPM 2024

LifeSciVC

JANUARY 16, 2024

Tirzepatide is also trialing in a Phase 3 obese HFpEF trial, and top-line readout in 2H 2024 will suggest whether the increased weight loss achieved with dual GLP1/GIPR modulation further improves QoL and functional endpoints. ALT) continue to build momentum in the indication. Will incretins “solve” MASH in obese patients?

Small Molecule

Small Molecule Trials Therapies Disease

#ScienceSaturday: January 20, 2024

KIF1A

JANUARY 20, 2024

Instead, this is what is called a splicing mutation; it occurs at the boundary between coding DNA that is read into RNA and protein, and noncoding DNA that is excluded. These boundaries need to be “spliced” or specifically cut so RNA and proteins only contain coding DNA.

DNA

DNA RNA Disease Research

Using a Function-First “Scout Fragment”-Based Approach to Develop Allosteric Covalent Inhibitors of Conformationally Dynamic Helicase Mechanoenzymes

Covalent Modifiers

DECEMBER 23, 2023

Kapoor Journal of the American Chemical Society 2024 DOI: 10.1021/jacs.3c10581 3c10581 Helicases, classified into six superfamilies, are mechanoenzymes that utilize energy derived from ATP hydrolysis to remodel DNA and RNA substrates. Chait, Ekaterina V. Vinogradova, and Tarun M.

RNA

RNA DNA Virus

Activity-Based Acylome Profiling with N-(Cyanomethyl)-N-(phenylsulfonyl)amides for Targeted Lysine Acylation and Post-Translational Control of Protein Function in Cells

Covalent Modifiers

SEPTEMBER 30, 2024

Abbasov Journal of the American Chemical Society 2024 DOI: 10.1021/jacs.4c09073 We identify paralog-selective chemical probes that acetylate conserved lysines within interferon-stimulated antiviral RNA-binding proteins, generating de novo proteoforms with obstructed RNA interactions. Ryan, Michael A. Norinskiy, Amy K.

RNA

RNA Drugs

Single Cell RNA Profiling FFPE Tissue

Fierce BioTech

NOVEMBER 5, 2024

Single Cell RNA Profiling FFPE Tissue Explore the robust cross-platform agreement between scRNA-seq and bulk RNA-seq, ensuring consistent and reproducible data on immune cell presence. png Tue, 11/05/2024 - 17:10 Biotech Fierce Biotech Homepage Landing Page Url [link] Byline Sponsored by: IQVIA Laboratories

RNA

RNA Laboratories

Tensor factorization + single-cell rna-seq

Broad Institute

AUGUST 8, 2024

Tensor factorization + single-cell rna-seq By lcopelan@broadinstitute.org August 8, 2024 Tensor factorization + single-cell rna-seq Neriman Tokcan UMass Boston MIA About Current Schedule Video Playlist Resources All talks Contact us MIA Talks Search - Any - Fall 2024 Spring 2024 Fall 2023 Spring 2023 Fall 2022 Spring 2022 Fall 2021 Spring 2021 Fall (..)

RNA

New diagnostic approach for bacterial infections shows promise in the clinic

Broad Institute

OCTOBER 21, 2024

By Leah Eisenstadt October 21, 2024 Credit: Kevin Middleton, Broad Communications In the Bhattacharyya lab, researchers develop methods to quickly identify the most appropriate treatments for bacterial infections. October 21, 2024. Paper cited Young EL, Roach DJ, et al. J Clin Micro.

RNA

RNA Hospitals Laboratories Medical Schools

Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Online January 8, 2024. By Sarah C.P. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

Engineering

Engineering Research Virus DNA

Simple test for flu could improve diagnosis and surveillance

Broad Institute

JUNE 18, 2024

Simple test for flu could improve diagnosis and surveillance By Allessandra DiCorato June 18, 2024 Breadcrumb Home Simple test for flu could improve diagnosis and surveillance A low-cost CRISPR-based paper strip test distinguishes between influenza types and can be reprogrammed to recognize different viruses including the H5N1 bird flu virus.

Virus

Virus RNA Laboratories Medical Schools

A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis

Broad Institute

APRIL 4, 2024

A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis By Rose Circeo April 4, 2024 Breadcrumb Home A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis The Primer on Medical and Population Genetics is a series of weekly lectures on genetics topics related to human populations and disease.

RNA

RNA Bioethics Disease Research

Streamlining the Path from Discovery to Patient – Simpler RNA Manufacturing with Aldevron’s Codex® HiCap RNA Polymerase

Fierce BioTech

JULY 1, 2024

Streamlining the Path from Discovery to Patient – Simpler RNA Manufacturing with Aldevron’s Codex® HiCap RNA Polymerase swheeler Mon, 07/01/2024 - 17:17 Wed, 08/07/2024 - 10:00 Resource Type Webinar Kyle Studey, M.S. On Demand Start Date Wed, 08/07/2024 - 11:00 Codex ® is a trademark of Codexis, Inc.

RNA

RNA Engineering Research

Fierce Pharma Forward: Drug Development and Delivery Insights for 2024

Fierce BioTech

APRIL 9, 2024

Fierce Pharma Forward: Drug Development and Delivery Insights for 2024 Join us for the inaugural Fierce Pharma Forward, a virtual event where industry leaders will explore the latest innovations in drug delivery and development. png Thu, 05/30/2024 - 00:05 Content Channel Landing Page Url LEARN MORE Byline May 30, 2024 | Free Virtual Event

Drug Development

Drug Development RNA Drugs

F. 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

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 By Corie Lok October 16, 2024 Breadcrumb Home F. By Broad Communications October 16, 2024 Credit: Erik Jacobs, Anthem Multimedia Richard N. Merkin (left) and F.

Vaccine

Vaccine RNA Laboratories Molecular Biology

#ScienceSaturday: February 3, 2024

KIF1A

FEBRUARY 4, 2024

We each have two DNA copies of the KIF1A gene; each copy is called an allele and makes its own RNA, which is then translated into protein. It is a type of gene-based therapy for KIF1A mutations that reduces RNA expression of the mutant allele, without interrupting healthy KIF1A expression. What is an allele specific ASO?

RNA

RNA DNA Disease Trials

To help his daughter living with an ultra-rare disorder, this dad brought together a squad of genetic detectives

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR. October 24, 2024.

DNA

DNA RNA Disease Hospitals

An immune factor in the brain plays critical roles in neuron function and aging

Broad Institute

JULY 19, 2024

By Boston Children's Hospital July 19, 2024 Credit: Scott-Hewitt N, et al. By Boston Children's Hospital July 19, 2024 Credit: Scott-Hewitt N, et al. Online June 27, 2024. DOI: 10.1016/j.cell.2024.05.058. 2024.05.058. C1q, produced by microglia (green and blue), interacts with neurons (purple and blue). DOI: 10.1016/j.cell.2024.05.058.

Protein Production

Protein Production Hospitals RNA Production

Machine learning guides researchers to new synthetic genetic switches

Broad Institute

OCTOBER 23, 2024

Machine learning guides researchers to new synthetic genetic switches By Tom Ulrich October 23, 2024 Breadcrumb Home Machine learning guides researchers to new synthetic genetic switches A new method allows precise activation or repression of genes in specific cells and tissues. Online October 23, 2024. By Sarah C.P.

Research

Research DNA Laboratories Molecular Biology

Some CRISPR screens may be missing cancer drug targets

Broad Institute

JUNE 14, 2024

Some CRISPR screens may be missing cancer drug targets By Allessandra DiCorato June 14, 2024 Breadcrumb Home Some CRISPR screens may be missing cancer drug targets Current CRISPR guides don’t work equally well in cells from people of all ancestries, which could lead to false negative results. Online June 7, 2024. Nature Communications.

Drugs

Drugs RNA DNA Research

A common fatty acid may help restore healthy vaginal bacteria after infection

Broad Institute

AUGUST 19, 2024

A common fatty acid may help restore healthy vaginal bacteria after infection By Ari Navetta August 19, 2024 Breadcrumb Home A common fatty acid may help restore healthy vaginal bacteria after infection Treatments using oleic acid, a naturally occurring oil used in cells, could improve treatment of recurrent bacterial vaginosis infections.

Hospitals

Hospitals Treatment Medical Schools Science

Schizophrenia and aging may share a common biological basis

Broad Institute

MARCH 6, 2024

Schizophrenia and aging may share a common biological basis By Allessandra DiCorato March 6, 2024 Breadcrumb Home Schizophrenia and aging may share a common biological basis Tightly synchronized genetic changes in two types of brain cells may underlie cognitive impairment in both conditions, offering potential therapeutic clues.

Medical Schools

Medical Schools RNA Science Research

New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor. Online May 16, 2024. Paper cited Huang Q, Chan KY, et al.

Therapies

Therapies FDA Approval Engineering Disease

New diagnostic approach for bacterial infections shows promise in the clinic

Broad Institute

OCTOBER 17, 2024

By Leah Eisenstadt October 17, 2024 Credit: Kevin Middleton, Broad Communications In the Bhattacharyya lab, researchers develop methods to quickly identify the most appropriate treatments for bacterial infections.

RNA

RNA Hospitals Laboratories Medical Schools

Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

SEPTEMBER 25, 2024

2022 Dec 12 [cited 2024 Sep 4];22(3):213–34. 2019 Dec 13 [cited 2024 Sep 4];122(4):465–72. 2020 Dec 1 [cited 2024 Sep 4];20(12):743–56. 2021 Oct 11 [cited 2024 Sep 4];13(20):5081. 2021 Apr [cited 2024 Sep 4];2(4):377–91. 2024 Mar 22 [cited 2024 Jul 8];84(6_Supplement):5891–1. Available from: [link] 2.Bell

Small Molecule

Small Molecule Therapies Regulations Treatment

NGS is evolving: collaboration and tech lead the way

Researchers map communities of single cells in metastatic breast cancers

Trending Sources

The biotech mapping thousands of hidden therapeutic clues

New tRNA tech aims to rewrite rare disease treatment

A spatial approach to understanding drug dynamics using mass spectrometry imaging

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development

Sanofi Showcases Rare Disease Pipeline Innovations at ISTH

A Visual Guide to Genome Editors

The Long Road to End Tuberculosis

In vivo CAR T: Faster, cheaper, and more effective cancer care

Targeting HER3 – a little wave of drug development that’s about to get a lot bigger

Lexeo to help launch spinout around RNA drugs for the heart

Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

Levers for Biological Progress

Atlas XIV And Reflections On The VC Math Problem

Optical density and absorbance measurements | BMG LABTECH

FDA’s Issues Draft Guidance on Accelerated Approval: A Substantial Evidentiary and Procedural Overhaul to this High-Profile Pathway

A covalent compound selectively inhibits RNA demethylase ALKBH5 rather than FTO

AZVUDINE

Blood Test Spots Early Pancreatic Cancers With 97% Accuracy

In Early Research, Heart Drugs Show Promise in Fighting Muscular Dystrophy

Sentiment & Themes Emerging From JPM 2024

#ScienceSaturday: January 20, 2024

Using a Function-First “Scout Fragment”-Based Approach to Develop Allosteric Covalent Inhibitors of Conformationally Dynamic Helicase Mechanoenzymes

Activity-Based Acylome Profiling with N-(Cyanomethyl)-N-(phenylsulfonyl)amides for Targeted Lysine Acylation and Post-Translational Control of Protein Function in Cells

Single Cell RNA Profiling FFPE Tissue

Tensor factorization + single-cell rna-seq

New diagnostic approach for bacterial infections shows promise in the clinic

Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Simple test for flu could improve diagnosis and surveillance

A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis

Streamlining the Path from Discovery to Patient – Simpler RNA Manufacturing with Aldevron’s Codex® HiCap RNA Polymerase

Fierce Pharma Forward: Drug Development and Delivery Insights for 2024

F. 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

#ScienceSaturday: February 3, 2024

To help his daughter living with an ultra-rare disorder, this dad brought together a squad of genetic detectives

An immune factor in the brain plays critical roles in neuron function and aging

Machine learning guides researchers to new synthetic genetic switches

Some CRISPR screens may be missing cancer drug targets

A common fatty acid may help restore healthy vaginal bacteria after infection

Schizophrenia and aging may share a common biological basis

New gene delivery vehicle shows promise for human brain gene therapy

New diagnostic approach for bacterial infections shows promise in the clinic

Looking beyond traditional oncogenic pathways to break cancer resistance

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