BioPharma Drive: Drug Pricing

APRIL 22, 2024

billion, is the latest research collaboration involving small molecule drugs that modify RNA. The deal, which could be worth up to $1.8

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Covalent Modifiers

FEBRUARY 25, 2024

ALKBH5, an m6A demethylase, is a promising target, particularly for anticancer drug discovery. TD19 irreversibly modifies the residues C100 and C267, preventing ALKBH5 from binding to m6A-containing RNA. However, the development of selective and potent inhibitors of ALKBH5 rather than FTO remains challenging.

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BioPharma Drive: Drug Pricing

JUNE 3, 2024

The alliance hands Lilly an RNA-based therapy in preclinical testing for ALS and frontotemporal dementia, and includes a research collaboration to develop other, similar medicines.

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BioPharma Drive: Drug Pricing

OCTOBER 9, 2023

The agency rejected Alnylam’s application for approval of its medicine patisiran in people with a rare heart condition, setting back the company’s plans.

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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.

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BioPharma Drive: Drug Pricing

JANUARY 23, 2024

J&J and Bristol Myers joined the latest funding round for the startup, which was led by the newly launched Mirae Asset Capital Life Science.

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BioPharma Drive: Drug Pricing

NOVEMBER 28, 2023

Tuesday’s deal signals the pharmaceutical giant's confidence in Avidity Biosciences and its so-called antibody oligonucleotide conjugates.

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Chemical Biology and Drug Design

DECEMBER 13, 2023

This up-to-date commentary/perspective article sheds light on the revolutionary approaches designed to target the principal viral weapon “RNA” used by RNA viruses (e.g., the severe acute respiratory syndrome coronavirus 2 “SARS-CoV-2”) to infect humans and spread infections, the genomic RNA strands.

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BioPharma Drive: Drug Pricing

AUGUST 9, 2023

The Flagship-backed company claims its technology can create genetic drugs that work broadly to treat diseases caused by “stop” mutations.

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BioPharma Drive: Drug Pricing

JANUARY 17, 2024

The biotech is developing an RNA-based therapy for what it describes as a root cause of the pregnancy-related complication, which affects millions of women.

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Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

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Drug Discovery Today

MAY 10, 2021

A new approach to tackling viruses by targeting the ‘control centre’ in viral RNA could lead to broad spectrum anti-viral drugs and provide a first line of defence against future pandemics, according to new research at the University of Birmingham.

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Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. Figure 1: High-level workflow for early drug discovery Once the raw data has been gathered, the next step is to gain a thorough understanding of the data.

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Broad Institute

OCTOBER 30, 2024

Eventually, we hope that these methods can help inform the development of drugs or even guide patients’ individual treatments.” In the new work, the team used two single-cell RNA sequencing methods to analyze 67 metastatic breast cancer biopsies collected from 60 different patients.

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Covalent Modifiers

SEPTEMBER 30, 2024

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.

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Drug Target Review

APRIL 7, 2025

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

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Elrig

MARCH 25, 2025

Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.

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Dark Matter Blog

APRIL 4, 2023

Prior to 2015, I had a casual relationship, at best, with targeting RNA. The bulk of my nearly three decades of experience up to that point was with drugging protein targets using a variety of modalities, but principally small molecules. Welcome to the RNA world. Familiar tools to solve familiar problems with proteins.

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Dark Matter Blog

AUGUST 31, 2020

The majority of small molecule drugs induce their therapeutic effects by seeking out and binding to their intended target while avoiding most other molecules in the dense milieu of the cell interior. Our overall mission at Arrakis is to expand the set of “druggable” targets for small-molecule medicines to include RNA.

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The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.

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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.

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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.

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Drug Discovery Today

MAY 13, 2021

INDIANAPOLIS and LONDON — Eli Lilly and Company (NYSE: LLY) and MiNA Therapeutics Limited, a pioneer in RNA activation therapeutics, today announced a global research collaboration to develop novel drug candidates using MiNA’s proprietary small activating RNA (saRNA) technology platform.

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Crown Bioscience

NOVEMBER 12, 2021

In this post, we explore how a targeted RNA-Sequencing (RNA-Seq) panel can be incorporated into preclinical drug development to evaluate the impact of immune effectors in mediating anti-tumor immunity of novel immuno-oncology (I/O) agents.

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Broad Institute

OCTOBER 21, 2024

Current methods for determining which drugs might work for each patient rely on growing bacteria from the patient in the lab and take days to yield results. In the meantime, patients are often given broad-spectrum antibiotics, which encourage drug-resistant infections, a significant public health threat.

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The Open Targets Blog

JANUARY 30, 2025

Amongst other tasks, gget opentargets can quickly find diseases and drugs associated with a specific gene. For example, to find drugs associated with the human IL13 gene (Ensembl ID ENSG00000169194*), a cytokine that plays an important role in allergic inflammation and immune response to parasite infection, you can use: # Install gget !

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Drug Target Review

AUGUST 30, 2023

Advancing drug discovery for pain disorders: human iPSC-based approach Drug discovery relies on the use of relevant model systems. Therefore, to advance drug discovery in the field, screens on human cellular models such as such as specialised sensory neurons (nociceptors) are necessary.

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Broad Institute

JANUARY 22, 2024

At the Broad Institute of MIT and Harvard and elsewhere, researchers are also learning how dependencies affect cancer cells and how they influence each other and contribute to drug resistance. Initially, there were two approaches: small hairpin RNA (shRNA)-based and now CRISPR-based experiments.

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Drug Hunter

JUNE 26, 2023

The program is notable as it’s a success for PTC outside of RNA splicing modulation, for which it has recently become famous thanks to the success of risdiplam and its emerging Huntington’s disease program. Drug Hunter Premium is drug discovery, distilled, so you can quickly catch up and make informed decisions based on industry examples.

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Broad Institute

AUGUST 31, 2023

We were surprised to see that just missing one copy of this gene can induce many changes at the RNA level, at the protein level, at the functional level, and at the behavioral level,” said Farsi. The scientists also found reduced levels of an enzyme that degrades dopamine, providing further evidence for dopamine’s role in the disorder.

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DrugBank

MAY 22, 2024

In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. A big part of the puzzle is selectivity—the ability of a drug to zero in on specific biological molecules without affecting others.

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Chemical Biology and Drug Design

AUGUST 24, 2023

In addition, the long-term administration of a single targeted drug can also lead to drug resistance and attenuate drug potency, so it is important to develop new targets for tumor therapy.

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Drug Target Review

JULY 5, 2023

In nature, proteins take millions and trillions of years to evolve, 1 but scientists do not have this time when designing drugs for life-threatening diseases. Therefore, researchers use directed protein evolution technologies , to evolve and design proteins with specific functions under well-defined conditions and a practical time frame.

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ASPET

SEPTEMBER 6, 2023

Toll-like receptor (TLR) 7 and TLR8 are single-stranded RNA-sensing endosomal pattern recognition receptors that evolved to defend against viral infections. However, aberrant TLR7/8 activation by endogenous ligands has been implicated in the pathogenesis of autoimmune diseases including systemic lupus erythematosus.

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DrugBank

MAY 15, 2024

The road to developing effective drugs is fraught with both promise and challenge, particularly when it comes to what scientists call "undruggable" targets. The Evolution of Drug Discovery: From Dark Rooms to Precision Targets In the past, drug discovery often felt like a lucky guess.

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Chemical Biology and Drug Design

DECEMBER 28, 2023

The prognostic value of long noncoding RNA (lncRNA) LINC00460 has been reported in cervical cancer. The interaction between miR-338-3p and LINC00460 or transforming growth factor β-induced protein (TGFBI) was confirmed by RNA immunoprecipitation (RIP) and luciferase reporter assays.

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Chemical Biology and Drug Design

JULY 27, 2023

cells induced by poly (I:C) were treated with or without PA, and then RNA-sequencing, GO and KEGG analysis were carried out to obtain the five differentially expressed genes (IL-1β, HMOX1, PTGS2, Abca1, and P65), which were verified by QRT-PCR and western blot. RNA-sequencing was applied to analyze the differentially expressed genes (DEGs).

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