Codon

APRIL 14, 2025

By creating competitive events for protocol optimization and “speedrunning challenges,” we’d target “hackers” of all experience levels. If successful, such events would not only generate concrete improvements but also help shift the culture toward valuing speed.

Protein Expression

SugarCone Biotech

JULY 6, 2025

On this basis, and to drive use of the RNA sequencing technology that identifies NRG1 fusions, the drug was purchased by Partner Therapeutics. Drug development has also been hampered by serious adverse events, including Grade 3 or higher levels of neutropenia and interstitial lung disease. regulatory application in NSCLC.

Drug Development

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. You can unsubscribe at anytime. Recent biotech company acquisitions have put emerging schizophrenia treatments in focus.

RNA

BioPharma Drive: Drug Pricing

JULY 16, 2025

Aside from the Duchenne medicines it sells, Sarepta intends to focus resources moving forward on a group of RNA-based medicines it’s developing with Arrowhead Pharmaceuticals. You can unsubscribe at anytime. Among them are treatments for spinocerebellar ataxia, Huntington’s disease and facioscapulohumeral muscular dystrophy. Will RFK Jr.

Therapies

BioPharma Drive: Drug Pricing

JULY 10, 2025

Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Moderna COVID vaccine gets full approval for children The approval comes amid regulatory upheaval under HHS head Robert F. You can unsubscribe at anytime. TechTarget, Inc.s

Vaccine

LifeSciVC

DECEMBER 5, 2024

combinatorial chemistry, human genetics, functional genomics, mRNA, I/O, CRISPR, RNA editing, and many others). He’s been a leading light in the biotech venture field, investing in essentially every major innovation wave of the last few decades (e.g.,

Marketing

Codon

AUGUST 4, 2025

Protein alterations, including phosphorylation and ubiquitination, can alter a protein’s function in ways that are invisible at the level of DNA or RNA. Scientists have already sequenced the RNA molecules of millions of brain cells from people with Alzheimer’s.

Laboratories

Twist Bioscience

APRIL 3, 2025

Human Comprehensive Exome Human Core Exome Human Methylome Panel Human RefSeq Panel Mitochondrial Panel Mouse Exome Panel Respiratory Virus Research Panel Comprehensive Viral Research Panel Twist's Whole Exome Library Preparation Workflow.

RNA

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

FDAs Overview of When Accelerated Approval is Appropriate The new draft guidance explains, that there are two circumstances in which the accelerated approval authority would most clearly apply: (1) when the course of the disease is long or (2) the clinical event that is relevant for demonstrating benefit occurs infrequently.

FDA

Conversations in Drug Development Trends

APRIL 15, 2025

The amyloid-tau hypothesis remains the prevailing model of AD pathogenesis, proposing that the accumulation of amyloid-beta (A) initiates a cascade of pathological events, with subsequent tau hyperphosphorylation and aggregation contributing to synaptic dysfunction, neuroinflammation, and neuronal loss.

Clinical Trials

Codon

JULY 21, 2025

There are only 5 nucleotide bases behind all life: adenine (A), cytosine (C), guanine (G) and thymine (T) in DNA, and uracil (U) taking the place of thymine in RNA. We’ve known about base pairs, RNA, and DNA for decades, but we couldn't make full use of them because our tools were so poor. This is a problem.

Science

New Drug Approvals

JULY 18, 2025

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

Drug Target Review

JANUARY 2, 2024

Forging the toolkit for subcellular omics Cellular components known as organelles, consisting of RNA and protein, exert vital influences on human wellbeing and ailments by sustaining equilibrium, managing growth and ageing, and producing energy.

RNA

Dark Matter Blog

APRIL 4, 2023

Prior to 2015, I had a casual relationship, at best, with targeting RNA. Targeting RNA is a Whole New World Then in 2015, I became smitten and eloped with RNA, setting out to build a company devoted to bringing to bear industrial drug discovery concepts and methods on a new problem of drugging RNA with small molecules.

Small Molecule

PLOS: DNA Science

JULY 13, 2023

Although the researchers followed all of these events with color-labeled DNA, the details of how the cells at the injury site are reprogrammed into a state of stemness are still a mystery. Analyzing RNA Revealed the Regeneration The researchers analyzed RNA to reconstruct the steps to regeneration. Do humans have similar genes?

RNA

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.

RNA

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. drug delivery development costs Resource Type Questex Event Fierce Pharma 250x190.png

Drug Development

Dark Matter Blog

JUNE 21, 2023

Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.

Small Molecule

The Pharma Data

OCTOBER 27, 2020

“Applying 3a’s proprietary enhanced RNA technology to PCR testing was a logical next step in our product development pipeline,” said Dr. Heinrich Jehle, managing director of 3a-Diagnostics. “We are currently developing two saliva-based tests to detect SARS-CoV-2 RNA.

RNA

The Pharma Data

DECEMBER 14, 2020

Supported by promising phase 1 data, CureVac announced Monday that it has enrolled the first participant in the pivotal phase 2b/3 study of its messenger-RNA based coronavirus vaccine, CVnCoV. No adverse events were observed and the reactions were similar to those seen in recovered COVID-19 patients, CureVac said ( DID , Nov.

Vaccine

The Pharma Data

MARCH 8, 2021

Week 96 findings reinforce the primary (proportion of participants with plasma HIV-1 RNA ?50 50 c/mL at Week 48 (Snapshot, ITT-E)), and secondary endpoints (proportion of participants with plasma HIV-1 RNA ?50 Week 48 primary endpoint (proportion of participants with plasma HIV-1 RNA ?50

RNA

The Pharma Data

MARCH 8, 2021

Of 202 treated participants, no safety signals have been identified and of the 4 serious adverse events reported, none were considered to be study drug related. In addition to the ongoing clinical studies, Merck has conducted a comprehensive nonclinical program to characterize the safety profile of molnupiravir. About Molnupiravir.

Trials

Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I wanted to use molecular biology to create drugs.

Small Molecule

The Strateos Blog: Drug Discovery

DECEMBER 13, 2021

SynBioBeta is one of synthetic biology’s premier events, bringing together a diverse community of thought leaders, technology innovators, engineers, scientists, and policy makers to explore the exciting new advancements that are shaping the future of science. Strateos’ own Daniel Rines, Ph.D.,

Engineering

The Pharma Data

OCTOBER 14, 2020

Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. About Eloxx Pharmaceuticals. Eloxx Pharmaceuticals, Inc. Eloxx Pharmaceuticals, Inc.

Pharmaceuticals

LifeSciVC

JANUARY 16, 2024

Still may be some merit if they are clinical-stage and can achieve once-daily dosing without too steep a cut in efficacy or worsened adverse events vs. peptides, but be wary of the intensely competitive landscape. A permanent gene edit to produce a difficult-to-titrate supply of GLP1 in the liver?

Small Molecule

The Pharma Data

SEPTEMBER 9, 2020

Immunization of non-human primates (rhesus macaques) with BNT162b2, a nucleoside-modified messenger RNA (modRNA) candidate that expresses the SARS-CoV-2 spike glycoprotein, resulted in strong anti-viral effects against an infectious SARS-CoV-2 challenge. The study is an event-driven trial. Argentina and Brazil.

Vaccine

Vial

DECEMBER 5, 2023

Among others, NGS has led to the identification of disease-causing variants and novel drug targets and an improved understanding of complex biological events, e.g., the heterogeneity of tumors. It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development.

Clinical Trials

Broad Institute

JULY 11, 2024

IDH1 normally facilitates the activity of enzymes called demethylases, which remove chemical flags called methylation marks from DNA, allowing genes to be transcribed into RNA. In cells with mutant IDH1, this gene is silenced due to the buildup of 2HG, but mIDH1 inhibitors allow demethylases to facilitate cGAS expression.

Treatment

The Pharma Data

DECEMBER 3, 2020

No serious adverse events related to the vaccine candidates studied were reported. The majority of adverse events were mild and transient. Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope.

Vaccine

DrugBank

JANUARY 29, 2025

Gene therapy strategies targeting oncogene inactivation, such as RNA interference and gene-editing technologies, have shown promise in preclinical and clinical studies. The system comprises a guide RNA that targets a specific DNA locus and the Cas9 enzyme that cleaves the DNA at the target site.

Therapies

The Pharma Data

JANUARY 31, 2021

No serious adverse events related to the vaccine candidates studied were reported. Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. The candidate vaccines were given in two doses, three weeks apart.

Clinical Trials

PLOS: DNA Science

MARCH 28, 2024

One type of histone addition, acetyl groups, bind in patterns that control which genes are expressed (transcribed into RNA and translated into protein) and which genes are hidden, a little like reading only some of the words on a page. Duvyzat reigns in HDAC activity, slowing the cascade of events leading to muscle deterioration.

FDA Approval

The Pharma Data

MAY 31, 2022

Olpasiran is a small interfering RNA designed to lower the body’s production of apolipoprotein(a), a key component of Lp(a) that has been associated with an increased risk of cardiovascular events.

RNA

PLOS: DNA Science

MARCH 7, 2024

That event blocked synthesis of the protein that signals formation of the tailbone. An Alu repeat is a “retrotransposon,” a short sequence of RNA that is copied into DNA that then inserts into the genome. Retrotransposons increase in number over time as DNA copies are peeled off of RNA templates.

DNA

The Pharma Data

AUGUST 19, 2021

Lenacapavir was generally well-tolerated, with no serious adverse events related to study drug and no study drug discontinuations through the 14-day period, including no discontinuations due to adverse events. The most common adverse events observed were injection site reactions.

Marketing

Drug Target Review

AUGUST 24, 2023

Infiltration of immune cells into tissues follows a cascade of events, namely rolling, activation, adhesion and transmigration, which are mediated by different molecules. How does GDF-15 impact the tumour microenvironment and the infiltration of T cells into the tumour? in North America) in late 2017.

Treatment