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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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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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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Pharmaceutical Technology

JUNE 16, 2023

from Flanders Innovation & Entrepreneurship (VLAIO) to further advance its oncology portfolio targeting RNA. It will also support the firm’s preclinical effort on the long non-coding RNA (LncRNA) programme, FTX-001, that targets MALAT-1. Flamingo Therapeutics has received a research grant of €1.7m

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Pharmaceutical Technology

AUGUST 6, 2024

SK pharmteco has entered an MoU with South Korea-based Rznomics for the development and manufacture of RNA-based gene therapies.

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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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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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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

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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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. The company’s VoyageR AI platform integrates computational methods to drug traditionally undruggable RNA targets.

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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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AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.

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Pharmaceutical Technology

DECEMBER 23, 2022

This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

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BioSpace

SEPTEMBER 22, 2020

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

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BioSpace

JUNE 12, 2022

Shape Therapeutics is evolving programmable RNA medicines that push the limits of current gene editing capabilities. BioSpace spoke with CSO David Huss.

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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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Pharmaceutical Technology

JANUARY 19, 2023

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. This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients.

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Pharmaceutical Technology

AUGUST 15, 2024

Clinical trials for the two RNA interference (RNAi) programmes are slated to commence in early 2025.

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Pharmaceutical Technology

JUNE 8, 2023

DTx-1252 is a fatty acid ligand conjugated oligonucleotides (FALCON) small interfering RNA (siRNA) therapeutic which represses the PMP22 gene in Schwann cells. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. An estimated 150,000 patients in Europe and the US are living with CMT1A.

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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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Bio Pharma Dive

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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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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BioPharma Reporter

JULY 4, 2024

RNA therapeutics have emerged as a transformative approach to treating many conditions, but the industry still typically relies on inefficient batch manufacturing methods.

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Pharmaceutical Technology

AUGUST 1, 2022

In March, the company entered a licensing agreement with Serum Institute of India (SII) to expedite access to messenger RNA products in emerging markets worldwide. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Topic sponsors are not involved in the creation of editorial content.

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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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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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BioPharma Reporter

OCTOBER 6, 2022

Ginkgo Bioworks is acquiring California biotech Circularis to strengthen its capabilities in cell and gene therapy.

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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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Scienmag

AUGUST 4, 2020

Led by Boyce Thompson Institute’s Andrew Nelson, four partners will identify RNA modifications and develop resources that may lead to hardier crops Credit: Photo credit: Anna Nelson Dittrich ITHACA, NY, August 4, 2020 — RNA perform a variety of functions in cells, helping with everything from regulating genes to building proteins.

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Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Clinical holds are becoming more common, especially in gene therapy programs.

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Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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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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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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BioSpace

SEPTEMBER 15, 2020

The company is genetically programming RNA not just to deliver a gene of interest, but to control the location, timing and intensity of therapeutic protein expression using mRNA-encoded logic circuits.

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Scienmag

APRIL 27, 2021

Credit: Nigel Michki Neurons result from a highly complex and unique series of cell divisions. For example, in fruit flies, the process starts with stem cells that divide into mother cells (progenitor cells), that then divide into precursor cells that eventually become neurons.

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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. Other initiatives include a Scientific Advisory Board that will help the firm establish its technology in RNA interference.

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