Drug Discovery World

DECEMBER 1, 2022

The consequence is the identification of drug targets that are not relevant to the biological process under study. . Join DDW for this free event, Broadening the horizon of drug discovery: use of unperturbed, native state cells to uncover true biological targets at single-cell level , which is supported by LevitasBio. Camila Egidio.

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pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

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Roots Analysis

NOVEMBER 6, 2023

RNA-based therapeutics have completely revolutionized the healthcare segment, greatly influencing the study and treatment of human diseases by conferring precise targeting ability to therapeutic modalities. The below figure presents the distribution of next generation RNA-based therapeutics and RNA-based vaccines, based on type of molecule.

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Drug Discovery World

DECEMBER 13, 2022

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . Using RNA interference, researchers restored the normal biological function of FKTN in patient-derived cells, offering hope for a new therapy. . Modifying RNA function.

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Drug Discovery World

AUGUST 1, 2023

Investigators at the Icahn School of Medicine at Mount Sinai have designed an RNA-based strategy to activate dendritic cells that eradicated tumours and prevented their recurrence in mouse models of melanoma. However, these have not been as successful in clinical trials as hoped.

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STAT News

JANUARY 30, 2023

This month marks the 40th anniversary of the Orphan Drug Act. That said, a lot has changed in science and drug development since 1983. It is now possible to treat diseases with gene therapy, antisense oligonucleotides, messenger RNA (mRNA), noncoding RNA (known as small interfering RNA, or siRNA), and other gene-based modalities.

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Drug Discovery World

DECEMBER 16, 2022

The investment allows the company to focus on discovering and developing novel small molecule therapies targeting RNA modifying enzymes (RMEs) for oncology and other diseases. . The post Biotech company raises $30M to target RNA modifying enzymes appeared first on Drug Discovery World (DDW).

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STAT News

JANUARY 9, 2023

An experimental RNA treatment reduced liver scarring in half of patients with an inherited disease called alpha-1 antitrypsin deficiency, or AATD, according to results from a mid-stage clinical trial reported Monday by its maker Arrowhead Pharmaceuticals.

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pharmaphorum

JANUARY 6, 2022

Roivant is the latest pharma group to take a position in the emerging field of therapies targeted at RNA splicing, licensing exclusive rights to a drug developed by a unit of Japanese drugmaker Eisai. . The post Roivant joins RNA splicing push with Eisai deal appeared first on.

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Pharmaceutical Technology

MAY 15, 2023

The US Food and Drug Administration (FDA) has given orphan drug designation to SiSaf’s siRNA [a double-stranded RNA molecule that is non-coding] therapeutic, SIS-101-ADO, for the treatment of autosomal dominant osteopetrosis type 2 (ADO2), a rare and serious skeletal disorder in children.

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Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields. News round-up for 13-16 May by DDW Senior Digital Content Editor Diana Spencer.

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pharmaphorum

OCTOBER 27, 2020

US biotech BioSig Technologies has abandoned a phase 2 trial of its antiviral drug merimepodib with Gilead’s Veklury in severe COVID-19 patients, after concluding the safety of the combination was in doubt. Moreover, as an orally-active drug merimepodib could also have found a role outside the hospital setting.

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Drug Discovery World

FEBRUARY 13, 2023

Dr Stephen Barat, Senior Vice President and Therapeutics Division Head at HTG Molecular Diagnostics tells DDW how transcriptomics is driving drug discovery and precision medicines. SB: Transcriptomics is the study of genomic RNA transcripts in a biological system. DDW: Could you explain the process of transcriptomics?

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pharmaphorum

OCTOBER 31, 2022

Verge Genomics has joined a select group of biotechs who have taken a drug discovered and developed using artificial intelligence into human testing. The post Verge Genomics takes AI-sourced drug for ALS into clinic appeared first on.

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Drug Discovery World

AUGUST 4, 2023

The pace of innovation in cancer drug discovery showed no signs of slowing this week, with several pretty momentous discoveries boosting research in the sector. The post This week in drug discovery (31 July – 4 August) appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

SEPTEMBER 16, 2022

The latest conditional recommendation is based on the final data from the SOLIDARITY clinical trial, which was sponsored by the WHO. These results are in line with those obtained from the placebo-controlled, double-blind ACTT-1 trial of the National Institute of Allergy and Infectious Diseases.

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Drug Discovery World

APRIL 3, 2023

Hosted by DDW and supported by Tecan, “How next-generation sequencing is informing oncology drug discovery” will be free to attend on April 24 at 7AM PST / 10AM EST / 3PM BST / 4PM CET. Join this free webinar to learn about the biggest challenges and opportunities facing the NGS oncology space. You will hear from Darrell Green, a lecturer in (..)

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Drug Discovery World

SEPTEMBER 15, 2023

Spanning artificial intelligence, theranostics, radiopharmaceuticals, quantum simulation and small regulatory RNA, the top stories this week all cover cutting-edge technologies and techniques making a huge impact on modern drug discovery and development.

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Drug Discovery World

APRIL 24, 2023

Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials?

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Drug Discovery World

APRIL 11, 2023

Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials?

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Drug Discovery World

APRIL 18, 2023

Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials?

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pharmaphorum

NOVEMBER 16, 2022

With cases of drug-resistant tuberculosis on the rise again around the world, GSK has sparked hopes of a new treatment option after reporting phase 2a data with a novel antitubercular drug for the disease. The post As resistant tuberculosis rises, GSK says trial backs novel antibiotic appeared first on.

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Pharmaceutical Technology

JANUARY 19, 2023

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). We look forward to working collaboratively with the FDA to bring the first RNA therapy directly targeting DUX4 to patients as quickly as possible.”

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Drug Discovery World

APRIL 4, 2023

The post Next-generation sequencing and oncology drug discovery appeared first on Drug Discovery World (DDW). Register now.

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Drug Discovery World

JANUARY 9, 2024

The company intends to use the proceeds to initiate and complete the first-in-human Phase I clinical trial and reach Phase II readiness for its lead program, RES-010, in obesity. The post €10M financing to support disease-modifying obesity drug appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. Results of that Phase I trial are expected to be presented later this year. The post Conversations from ESMO Targeted Anticancer Therapies Congress 2024 appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

JUNE 6, 2023

According to Globaldata, it is involved in 1 clinical trial, which is ongoing. The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). The drug candidate is administered by intravenous route.

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The Pharma Data

AUGUST 16, 2021

The antiviral agent incorporates RNA-like building blocks into the RNA genome of the virus. If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. Molnupiravir is currently being tested in clinical trials. ” Mutations in the genome stop the virus.

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Pharmaceutical Technology

AUGUST 2, 2022

GreenLight’s process from formulation of the drug substance and lipid nanoparticle to bulk drug product can be carried out in this facility. The RNA platform of Samsung Biologics permitted GreenLight to move from mRNA vaccine conceptualisation to the delivery of released clinical trial material in under two years.

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Pharma Mirror

FEBRUARY 2, 2022

Global contract development and manufacturing organisation (CDMO), Recipharm, has today announced the acquisition of GenIbet, a Portuguese CDMO, specialising in the manufacture of biological clinical trial material and novel modalities such as viral vectors, RNA and microbiome.

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Drug Discovery World

MARCH 21, 2023

Diana Spencer explores the different ways the drug discovery sector is tackling colorectal cancer. So, what are the recent developments and how is the drug discovery sector tackling this common cancer? Improved diagnostics Diagnostics and screening are essential to the early treatment of colorectal cancer.

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STAT News

SEPTEMBER 30, 2022

Participants in the study have undergone liver biopsies at baseline and one year to determine if fazirsiran improves disease symptoms, including liver fibrosis.

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Drug Discovery World

MAY 18, 2023

A clinical trial of an experimental universal influenza vaccine developed by researchers at the National Institute of Allergy and Infectious Diseases’ (NIAID) Vaccine Research Center (VRC) has begun enrolling volunteers at Duke University in Durham, North Carolina. The trial will enrol up to 50 healthy volunteers aged 18 to 49.

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pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

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Drug Discovery World

FEBRUARY 19, 2024

Replicate Bioscience has shared positive results from the Phase I trial of RBI-4000, its srRNA-based rabies vaccine. In this clinical trial, evaluating safety and immunogenicity of RBI-4000 , participants received one or two doses of srRNA vaccine at low doses (0.1mcg, 1mcg or 10mcg).

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Delveinsight

SEPTEMBER 23, 2021

Boehringer Ingelheim acquires Abexxa Biologics for precision most cancers drug pipeline. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. Stafford and co.

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pharmaphorum

SEPTEMBER 2, 2020

Spain’s Highlight Therapeutics is attempting to get patients with melanoma to respond to Keytruda by combining with its RNA-based cancer therapy in patients with melanoma who have progressed despite treatment with PD-1 therapies such as Keytruda or BMS’ rival Opdivo (nivolumab). A total of 40 patients are planned to be enrolled.

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