Drug Discovery World

SEPTEMBER 12, 2022

As the biopharmaceutical industry increases its focus on developing RNA therapeutics, the new RNA solution could help R&D organisations accelerate the delivery of drug candidates. The post New solution to boost RNA drug development appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

JUNE 8, 2023

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

AUGUST 3, 2023

Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G 2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. The post Théa acquires ProQR’s RNA ophthalmic assets appeared first on Drug Discovery World (DDW).

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Bio Pharma Dive

NOVEMBER 14, 2022

The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

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pharmaphorum

NOVEMBER 1, 2022

14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Date: 8 – 9 February 2023.

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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. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.

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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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BioTech 365

JUNE 28, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Outsmarting cancer with RNA, ‘genome-tuning’ drugs and other gene-altering therapies.Outsmarting cancer with RNA, ‘genome-tuning’ drugs and other gene-altering therapies arlene.weintraub Tue, 06/22/2021 (..)

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BioTech 365

MARCH 10, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Northwestern U’s novel gene-silencing RNA drug shows promise in glioblastoma.Northwestern U’s novel gene-silencing RNA drug shows promise in glioblastoma aliu Wed, 03/10/2021 – 06:56 from FierceBiotech: Biotech (..)

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Drug Discovery World

APRIL 11, 2023

US-based start-up Atomic AI is using artificial intelligence (AI) to design more intelligent RNA-targeted and RNA-based medicines. Artificial Intelligence (AI) has been enthusiastically adopted by the drug discovery sector as a tool for shortening drug discovery cycle times and increasing success rates.

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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

JANUARY 31, 2024

Ian Scanlon will discuss bottlenecks in advanced therapeutic delivery and how novel technologies can improve patient access and deliver sustainable processes.

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Drug Discovery World

FEBRUARY 15, 2024

Ian Scanlon will discuss bottlenecks in advanced therapeutic delivery and how novel technologies can improve patient access and deliver sustainable processes.

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Drug Discovery World

OCTOBER 10, 2022

RNA sequencing has become a widely used tool that can explore the transcriptome in a seemingly endless number of applications. The post Is single-cell gene expression the next trend in next generation sequencing? appeared first on Drug Discovery World (DDW). Join now for FREE – it takes less than 2 minutes! Username or E-mail.

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Drug Discovery World

FEBRUARY 22, 2024

Ian Scanlon will discuss bottlenecks in advanced therapeutic delivery and how novel technologies can improve patient access and deliver sustainable processes.

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Drug Discovery World

FEBRUARY 5, 2024

Ian Scanlon will discuss bottlenecks in advanced therapeutic delivery and how novel technologies can improve patient access and deliver sustainable processes.

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BioTech 365

DECEMBER 1, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Researchers identify key RNA “gatekeeper” in gene expression, pointing to possible new drug targets.An

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Pharmaceutical Technology

FEBRUARY 9, 2023

The collaboration is aimed at discovering and developing new advanced treatments for genitourinary (GU) tumours as well as other related serious diseases using ReviR’s RNA-targeted technologies. ReviR combines computational and high throughput drug discovery technologies to deliver advanced medicines to patients.

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Drug Discovery World

FEBRUARY 27, 2023

DDW’s Diana Spencer speaks to Raphael Townshend PhD, Founder and CEO of Atomic AI, about the company’s successes so far, plans for the future and the unique approach of combining RNA structural biology and AI models. DS: How is Atomic AI “unlocking the next generation of RNA drug discovery”? What is unique about your engine?

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Drug Discovery World

FEBRUARY 13, 2024

In this webinar, hosted by DDW and sponsored by Astrea Bioseparations, you will learn about how market growth can be sustained to maximise on opportunities in cell and gene therapy (CGT). Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor.

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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.

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Drug Discovery World

JANUARY 10, 2023

This collaboration aims to further exploit circRNAs by developing a novel mechanism to facilitate RNA circularisation specifically in colorectal cancer cells. The post Alliance to develop circular RNA-based therapeutics appeared first on Drug Discovery World (DDW).

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Drug Discovery World

AUGUST 16, 2023

Samir Ounzain , PhD, CEO & Co-Founder of HAYA Therapeutics, looks at how a better understanding of our DNA can lead to increased activity for RNA therapeutics. The whole world realised the power of RNA when the Covid-19 pandemic brought us the first mRNA-based vaccines.

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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

JUNE 13, 2024

Importantly, our work has pinpointed that metformin uses small pieces of RNA (called microRNAs) to act as a ‘circuit breaker’ and turn off certain genes that are involved in cell growth and division, so it is possible that our findings could eventually be used to develop a new targeted cancer therapy. “In

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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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Drug Discovery World

JANUARY 27, 2023

Cell and gene therapies have been making the news this week, as we launch our very own guide to global innovation in CGT with the Winter issue of DDW, as well as an exciting new virtual event on the same topic. The post This week in drug discovery (23-27 January) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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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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Drug Discovery World

NOVEMBER 1, 2022

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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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). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4.

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Drug Discovery World

OCTOBER 14, 2022

The top news in the world of drug discovery this week reflects the vital importance of collaboration, partnership and support within this industry. . Gene editing platform could shape future application of CRISPR-based therapeutics . 30 million financing for RNA therapies for chronic liver diseases .

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pharmaphorum

OCTOBER 13, 2020

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. According to the company it will look to develop drugs that work against multiple targets that influence disease.

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Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery.

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Medical Xpress

MAY 16, 2023

This is the deactivation of the NSUN7 gene, which is an epigenetic editor of RNA. Tumors with this alteration tend to have a poor clinical prognosis, although the research indicates that they are more vulnerable to bromodomain inhibitors, a family of anticancer drugs.

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