PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Muscle makes up about 40 percent of body weight. million DNA bases.

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FDA Approval Treatment FDA DNA 97

Conversations in Drug Development Trends

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness.

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DrugBank

JULY 24, 2024

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

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Small Molecule Drugs Clinical Trials Therapies 82

Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

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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.

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Small Molecule Molecular Biology Drugs Disease 81

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline.

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Small Molecule Trials Therapies Disease 138

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).

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Clinical Trials Trials RNA Bioinformatics 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.

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RNA Cell Biology Clinical Development DNA 64

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 suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life.

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Engineering Virus DNA Therapies 137

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.”.

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RNA Pharmaceuticals Virus Clinical Trials 52

The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Silverback Therapeutics

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RNA Therapies Disease Protein Expression 52

Drug Target Review

MAY 27, 2024

Caris Discovery, the therapeutic research arm of Caris Life Sciences, was launched to address the paucity of novel oncology drug targets and is uniquely poised to discover first-in-class ADC targets. What role do ADCs play in Caris’ strategy for targeted cancer therapy, particularly in terms of patient-specific treatment plans?

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Clinical Trials Science Molecular Biology Bioinformatics 59

Drug Target Review

SEPTEMBER 18, 2024

What specific cost-cutting measures can AI provide in the manufacturing process of cell and gene therapy, and how do these compare to traditional methods? The cell and gene therapy (CGT) space provides a great example of the potential of AI. AI can vastly improve cell culture, a critical step in cell therapy production.

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Therapies Doctors RNA Production 52

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.

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RNA Small Molecule Synthetic Chemistry Science 52

The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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Clinical Trials RNA DNA Science 52

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.

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RNA Disease Trials Therapies 52

The Pharma Data

DECEMBER 13, 2020

Their extensive experience and proven track record in advancing innovative therapies, in addition to strong leadership skills, will help us to strengthen our portfolio and accelerate technology translation to help patients in need.”.

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Medical Schools Clinical Development Therapies RNA 40

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.

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Vaccine Immune Response Virus RNA 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.

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Therapies Virus Treatment Clinical Trials 40

The Pharma Data

NOVEMBER 24, 2020

Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. Until today, there were no approved pharmaceutical therapies for PH1.

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FDA Approval Treatment FDA RNA 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. We’re the middleman,” he said.

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Disease Clinical Trials Small Molecule Treatment 52

SugarCone Biotech

MAY 28, 2019

Lymphoid Structures Drive Immune Checkpoint Therapy and the Efficacy of Cellular Therapeutics (a reboot from 2020) We’d been hearing the rumors for months. Further in a small cohort of patients (n=47), classes E and D, those most likely to have TLS, responded best to ICB therapy. So, are these observations generalizable?

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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. Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro. ” The findings published today (Feb.

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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.

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Drug Development Clinical Pharmacology Immune Response Drugs 52

Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

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Conversations in Drug Development Trends

MAY 30, 2024

This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. The shared placebo design is another approach to accelerating drug development in ALS research.

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PPD

NOVEMBER 8, 2022

As 2022 comes to a close, we’re reflecting on the past 12 months as a transformative year for the drug development industry — one that foreshadows both change and opportunity in biopharma and biotech clinical trials in 2023. Six Predictions for the Drug Development Industry in 2023 1.

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Clinical Trials Trials Drug Development Clinical Research 52

The Pharma Data

AUGUST 29, 2020

Inclisiran is currently under review by the FDA and the EMA for the treatment of primary hyperlipidemia (including Heterozygous Familial Hypercholesterolemia) in adults who have elevated LDL-C while being on a maximally tolerated dose of statin therapy. Global Head of Cardiovascular, Renal and Metabolic Drug Development, Novartis.

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Treatment Disease Therapies Trials 52

The Pharma Data

JANUARY 31, 2021

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. About CpG 1018.

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Vaccine Trials Virus RNA 52

The Pharma Data

JANUARY 31, 2021

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. About Dynavax.

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Clinical Trials Vaccine Trials Virus 52

The Pharma Data

SEPTEMBER 8, 2020

About AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.

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The Pharma Data

SEPTEMBER 8, 2020

(LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Cambridge, UK.

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Science Vaccine Clinical Trials Pharmaceutical Companies 52

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

The new draft guidance , Expedited Program for Serious Conditions Accelerated Approval of Drugs and Biologics , is intended to replace much of the 2014 Guidances discussion of the topic. However, the 2014 Guidance cannot be ignored entirely. However, while FDA reiterated its interpretation that the statutory requirement to take into account.

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Drug Target Review

NOVEMBER 27, 2024

Introduction Biomarkers are becoming increasingly essential in drug development and clinical practice, driving the need for more precise validation methods. Given this, what’s holding drug developers back? Advanced techniques like MSD and LC-MS/MS offer unmatched precision and sensitivity at a fraction of the cost.

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Drug Target Review

FEBRUARY 4, 2025

Currently, there is no cure, and available treatments can only temporarily alleviate symptoms, making the need for new, effective therapies more urgent than ever. These innovative therapies represent a new frontier in Alzheimers care, addressing the critical need for effective treatment options.

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Codon

FEBRUARY 5, 2025

Cynomolgus macaques, small primates native to Southeast Asia, are already widely used in drug development research. The researchers also ran follow-up experiments in the laboratory; cell lines carrying the same mutations as those found in the monkey had similar RNA expression patterns as cells taken from Parkinson’s patients.

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