Pharmaceutical Technology

JULY 29, 2022

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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Bio Pharma Dive

JULY 17, 2023

For $500 million, Novartis will acquire DTx Pharma and its preclinical neurological disease drugs, marking the Swiss company’s latest investment in gene-silencing medicines.

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Pharmaceutical Technology

APRIL 3, 2023

The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.

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STAT News

NOVEMBER 14, 2022

Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA.    On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.

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Pharmaceutical Technology

OCTOBER 11, 2022

Biotechnology company Ochre Bio has raised $30m in a Series A financing round to develop RNA therapies for chronic liver diseases. It also intends to turn the insights from its research into RNA-based drug candidates, and they will be tested in human livers at the company’s recently opened ‘Liver ICUs’ in the US.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Pharmaceutical Technology

MARCH 22, 2024

is revolutionizing Parkinson's disease treatment with RNA molecules and CRISPR technology. Discover how AnGes Inc. Learn about their patent for precise genetic modifications.

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.

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Scienmag

JULY 19, 2022

A key genetic mutation that occurs early on in cancer alters RNA “dark matter” and causes the release of previously unknown RNA biomarkers for cancer early detection, a new study by UC Santa Cruz researchers published in the journal Cell Reports shows. Credit: Daniel H. Kim A key genetic mutation that occurs early on in […].

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BioSpace

DECEMBER 13, 2020

San Diego-based Locanabio secured $100 million in a Series B financing round that will be used to advance the company’s portfolio of novel RNA-targeted gene therapies for neurodegenerative, neuromuscular and retinal diseases.

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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

FEBRUARY 28, 2023

An alliance with the startup brings Vertex an RNA-based medicine for the disease, adding to a portfolio that already includes a gene editing treatment.

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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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Scienmag

MARCH 9, 2021

CHOP researchers find small changes in RNA due to RNA editing could have a large impact on gene expression and phenotype Philadelphia, March 9, 2021–Combining computational mining of big data with experimental testing in the lab, researchers at Children’s Hospital of Philadelphia (CHOP) have identified RNA editing events that influence (..)

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Scienmag

JUNE 13, 2022

New research from UT Southwestern suggests that RNA exosomes – the cellular machines that degrade old molecules of RNA – play a key role in the development of B cells, which are critical to the immune system’s ability to protect against infection.

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Bio Pharma Dive

AUGUST 18, 2022

The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.

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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

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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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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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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BioPharma Reporter

JANUARY 23, 2023

Charles River Laboratories International, Inc and Rznomics Inc, a South Korea-based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, have established a viral vector contract development and manufacturing organization (CDMO) partnership.

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Pharmaceutical Technology

JANUARY 29, 2024

An eye-catching milestone in hand, Ascidian will begin the Phase I/II STELLAR study of ACDN-01 for Stargardt disease in H1 2024.

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Pharma Times

OCTOBER 21, 2021

Givlaari uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people AHP

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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. As tech experts call for improved regulation and a halt to the development of AI, we take a look at how one company, Atomic AI, is using it to develop RNA-targeting treatments for a range of diseases.

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BioSpace

SEPTEMBER 22, 2020

RNA-binding proteins are emerging as the next generation of molecular medicine.

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Drug Discovery World

FEBRUARY 15, 2024

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

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

FEBRUARY 22, 2024

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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BioSpace

SEPTEMBER 11, 2020

I certainly can’t, but Eclipse BioInnovations, a San Diego-based biotech start-up, is aiming to do just that for RNA therapeutics. Can you imagine organizing more than 10 terabytes of data into a useful network of information, like creating Google maps from scratch?

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Drug Discovery World

JANUARY 31, 2024

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

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? While bulk sequencing—or sequencing the transcriptome of an entire sample at once—has contributed to many [.]. Remember Me.

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pharmaphorum

APRIL 25, 2022

Are you looking to solve challenges with current RNA therapeutics, such as the degradation of RNA by RNAses, the difficulty of RNA crossing the cell membrane by diffusion and the immunogenicity elicited by exogenous RNA? Next generation RNA therapeutics can help! Take a look at the full event guide to learn more!

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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. This landscape is rapidly changing.

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Drug Discovery World

FEBRUARY 5, 2024

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

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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BioSpace

JULY 14, 2021

The amount is intended to support the company's growing portfolio of RNA technologies and accelerate efforts to develop various treatment approaches in the gene therapy field.

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