DrugBank

MAY 22, 2024

Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins. DNA and RNA are also key players, each bringing unique challenges. The ultimate goal is to develop molecules that can interact with DNA without damaging it. RNA, with its diverse functions and structures, offers a new frontier in drug design.

Small Molecule 66

Small Molecule RNA DNA Pharmaceutical Companies 66

DrugBank

MAY 15, 2024

Its structure makes it incredibly difficult for drugs to bind effectively, which has stymied drug development for decades. The binding pockets on KRAS are shallow and polar , not ideal for the kinds of interactions needed for strong, effective drug binding.

Small Molecule 81

Small Molecule Molecular Biology Drugs Disease 81

The Pharma Data

OCTOBER 21, 2020

AT-527 is expected to be ideally suited to combat COVID-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses. Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.”.

RNA 52

RNA Pharmaceuticals Virus Clinical Trials 52

Drug Target Review

APRIL 9, 2024

In a previous study, we showed that these peptides decorate all cellular RNAs and thus impair the binding of RNA-binding proteins to RNA. Initial works from the lab concentrated on exploring the role of replicative stress in cancer and ageing, for which the group combined cell biology, mouse models and drug development projects.

RNA 64

RNA Cell Biology Clinical Development DNA 64

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

Therapies 52

Therapies DNA RNA Laboratories 52

NIH Director's Blog: Drug Development

SEPTEMBER 29, 2015

Next, to see how their tiny models of the human brain respond to various chemicals, the researchers exposed the neural tissue chips to 60 compounds—about half with known neurotoxic effects, and the rest considered harmless to the brain—and measured the activity of 19,000 genes in each organoid by RNA sequencing.

RNA 52

RNA Science Laboratories Clinical Trials 52

Vial

DECEMBER 5, 2023

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

Clinical Trials 52

Clinical Trials Trials RNA Bioinformatics 52

Drug Target Review

SEPTEMBER 25, 2024

However, this will not be straightforward, given that non-oncogene resistance is driven by complex transcriptional networks, and the key drivers that determine how these mechanisms could be targets for drug development remain elusive.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

Dark Matter Blog

APRIL 8, 2020

Most people, and in particular most investors we spoke to in those early days, thought we were nuts – that RNA lacked the structural and molecular complexity that medicinal chemistry exploits with such great effect for proteins. One is that molecular recognition is purely a matter of physics and RNA has to play by the same rules.

RNA 52

RNA Small Molecule Synthetic Chemistry Science 52

Drug Target Review

MAY 27, 2024

By identifying proteins that only interact with each other in cancer, but not normal tissues, we can really expand the repertoire of ADC therapeutics beyond monoclonal antibodies developed based solely on RNA expression profiles.

Clinical Trials 59

Clinical Trials Science Molecular Biology Bioinformatics 59

Conversations in Drug Development Trends

AUGUST 14, 2024

Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways. However, at Worldwide, we have a proven track record of successfully utilizing biomarkers in oncology development.

Treatment 78

Treatment Therapies Disease Protein Expression 78

The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost.

RNA 52

RNA Therapies Disease Protein Expression 52

Codon

NOVEMBER 3, 2024

As an undergraduate biology student, I spent some time in a TB lab working on antibiotic resistance — a growing concern for drug developers. The researchers were left with 40 drug candidates. However, hope prevails.

Vaccine 92

Vaccine Trials Therapies Clinical Trials 92

PLOS: DNA Science

MARCH 28, 2024

It “could have been easily repurposed for DMD,” said Pier Lorenzo Puri at Sanford Burnham Prebys Medical Discovery Institute in San Diego, who has been working on the drug development since 2004. MDA supported development of a mouse model that enabled the preclinical studies of what would become Duvyzat.

FDA Approval 97

FDA Approval Treatment FDA DNA 97

DrugBank

JULY 24, 2024

This was the first successful drug development against a transcription factor, opening the door to targeting a whole new class of previously unreachable targets. The Promise of Multispecific Drugs The future of drug discovery looks even brighter with the development of multispecific drugs.

Small Molecule 82

Small Molecule Drugs Clinical Trials Therapies 82

The Pharma Data

OCTOBER 19, 2020

The company’s suite of market-leading molecular profiling offerings assesses DNA, RNA and proteins to reveal a molecular blueprint that helps physicians and cancer patients make more precise and personalized treatment decisions.

Clinical Trials 52

Clinical Trials RNA DNA Science 52

The Pharma Data

DECEMBER 3, 2020

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. Trimer-Tag © is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins.

Vaccine 52

Vaccine Immune Response Virus RNA 52

LifeSciVC

JANUARY 16, 2024

Whether trial design, execution, or otherwise, drug development even where there is precedent is a challenging road and should not be taken for granted. Finally, I would be remiss if I didn’t caveat that we still see many clinical failures even for “de-risked” biology, as nothing is ever truly low-risk in our industry.

Small Molecule 138

Small Molecule Trials Therapies Disease 138

Broad Institute

OCTOBER 11, 2023

The method prevents the mouse’s immune system from rejecting the CRISPR machinery while also leaving behind RNA barcodes that can be sequenced to determine which gene had been deactivated in each cell. “I

Research 137

Research Immune Response Clinical Trials Therapies 137

The Pharma Data

OCTOBER 25, 2020

The investments in this proprietary platform have enabled Moderna to expeditiously create, manufacture and clinically develop mRNA-1273 to potentially address the current COVID-19 pandemic. Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. 26, 2020 11:00 UTC.

Vaccine 52

Vaccine Clinical Development RNA Clinical Trials 52

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 ). It wasn’t a silver bullet to the hard problem of drug development, but it wasn’t too far off either. There was scFormer in 2022, scGPT in 2023, and plenty of others.

Engineering 137

Engineering Virus DNA Therapies 137

The Pharma Data

AUGUST 30, 2021

Global Head of Cardiovascular, Renal and Metabolic Drug Development, Novartis. “As As the first and only small interfering RNA to provide effective and sustained LDL-C reduction, Leqvio helps manage a critical cardiovascular risk factor for ASCVD. Food and Drug Administration (FDA) and other health authorities.

RNA 52

RNA Disease Trials Therapies 52

PerkinElmer

AUGUST 18, 2020

HTRF is a common in-vitro assay modality used in drug development. Amplified luminescent proximity homogeneous assays, or Alpha assays, measure response to non-radioactive fluorescent emission by tagged molecules located near the target molecule.

Virus 52

Virus Vaccine RNA DNA 52

The Pharma Data

DECEMBER 8, 2020

chief executive officer at Ionis, said, “In 2020, we pursued an aggressive agenda focused on building our commercial plans and capabilities, progressing the Ionis-owned pipeline, advancing our technology and growing our leadership position in RNA-targeted therapeutics. Monia , Ph.D., ” Highlights of Investor Day.

RNA 52

RNA Pharmaceuticals Disease Treatment 52

Elrig

APRIL 29, 2024

Harini joined Serna Bio in early 2021 and has been an integral part of the multidisciplinary teamworking on target ID platform development to drug discovery. She has played a key role inbuilding the target identification platform and a proprietary database of transcriptome-wide, functional RNA structures.

Bioinformatics 59

Bioinformatics Drugs Molecular Biology Cell Based Assays 59

The Pharma Data

DECEMBER 13, 2020

Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. Before that, he was Director of Development at Cobalt Biomedicine leading CAR-T and other cell/gene therapy programs, and R&D Director at OvaScience developing stem cell-based products.

Medical Schools 40

Medical Schools Clinical Development Therapies RNA 40

The Pharma Data

OCTOBER 28, 2020

Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today confirmed that the Ministry of Health, Labour and Welfare of Japan (MHLW) and Takeda Pharmaceutical Co., 29, 2020 06:00 UTC. CAMBRIDGE, Mass.–(

Vaccine 40

Vaccine Government Clinical Trials RNA 40

The Pharma Data

NOVEMBER 24, 2020

Oxlumo works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.

FDA Approval 52

FDA Approval Treatment FDA RNA 52

Drug Target Review

SEPTEMBER 18, 2024

Companies are planning their drug development strategies with automated unit operations in mind, and how to incorporate them earlier in the development pipeline. Throughout his career in the industry, Dalip has been engaged in developing technologies and methods for use in cell therapy applications.

Therapies 52

Therapies Doctors RNA Production 52

The Pharma Data

JANUARY 17, 2021

That’s not the case with RNA-targeted drugs. Traditional companies have neither the technologies nor the financial incentives needed to develop ultra-rare therapies. Working closely with Ionis, “We connect the need to the drug development engine. Crooke, therefore, created n-Lorem Foundation to do that charitably.

Disease 52

Disease Clinical Trials Small Molecule Treatment 52

NIH Director's Blog: Drug Development

NOVEMBER 16, 2021

Meanwhile, Britain recently approved the other recently developed antiviral molnupiravir, which slows viral replication in a different way by blocking its ability to copy its RNA genome accurately.

Clinical Trials 52

Clinical Trials Virus Hospitals FDA 52

The Pharma Data

FEBRUARY 25, 2022

” Previous research has demonstrated that two SARS-CoV-2 enzymes – proteases including Mpro and PLpro – are promising targets for antiviral drug development. According to Jose, these enzymes are relatively stable; therefore, they are unlikely to develop drug-resistant mutations rapidly.

FDA Approval 52

FDA Approval FDA Molecular Biology Virus 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

The Pharma Data

NOVEMBER 16, 2020

Moderna’s partner PPD (Nasdaq: PPD), a leading global contract research organization providing comprehensive, integrated drug development, laboratory and lifecycle management services, has also been essential to the successful execution of the COVE study. CAMBRIDGE, Mass.–(BUSINESS –(BUSINESS WIRE)–Nov.

Vaccine 52

Vaccine Clinical Trials FDA Disease 52

Molecule Blog

MARCH 17, 2023

For instance, RNA sequencing is highly sensitive to batch effects , and using a single high-quality RNAseq provider to federate data is more likely to result in the highest statistical power than multiple providers who don’t share data and correct each dataset for data science purposes.

Engineering 52

Engineering Research Clinical Trials Government 52

SugarCone Biotech

MAY 28, 2019

This broader and still evolving contextual framework will impact immunotherapy drug development across the immune checkpoint field, the tumor vaccine space, innate immune approaches, the T-cell-directed biologics, and cellular therapies. But first, these new papers are gorgeous: The study presented by Petitprez et al.

Immune Response 52

Immune Response Therapies Production Vaccine 52

The Pharma Data

OCTOBER 27, 2020

The collaboration aims to leverage Molecular Partners’ proprietary DARPin® technologies and Novartis’ broad expertise in global drug development, regulatory affairs, manufacturing and commercialization to rapidly advance the program in keeping with the unprecedented global urgency created by the pandemic.

Therapies 40

Therapies Virus Treatment Clinical Trials 40