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

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. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

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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

APRIL 27, 2023

Orbital Therapeutics has raised $270m in a Series A round led by ARCH Venture Partners to advance a portfolio of programmable RNA therapeutics. Orbital will use the new funding to increase the application of RNA-based medicines for use in the fields of new vaccines, immunomodulation and protein replacement.

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Pharmaceutical Technology

APRIL 20, 2023

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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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. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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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. Around 40% of clinical holds are for gene therapy programs.

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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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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

AUGUST 15, 2024

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

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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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BioSpace

SEPTEMBER 22, 2020

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

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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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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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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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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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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

MAY 19, 2023

We are pioneering the convergence of immunology and RNA science to build a clinical-stage portfolio of products, starting with the significant unmet needs of cancer patients. “We Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

DECEMBER 11, 2022

The first subject was dosed in the trial, which is designed to evaluate the safety, tolerability and immunogenicity of a nucleoside-modified RNA-based combination vaccine approach. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 5 Omicron sublineages spike proteins. By Cytiva Thematic.

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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

4/5 strain messenger RNA (mRNA). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The contract comprises a $1.74bn award to produce and supply these vaccine doses and options to further procure up to 234 million additional doses of the company’s booster candidates.

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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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XTalks

NOVEMBER 15, 2024

XTALKS WEBINAR: Solutions for Vaccine Innovation and Gene Therapy: Unlocking the Power of RNA Live and On-Demand: Tuesday, December 17, 2024 , at 9am EST (3pm CET / EU-Central) Register for this free webinar to explore how next-generation RNA technologies can provide effective solutions for vaccine innovation and gene therapy.

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Pharmaceutical Technology

JULY 15, 2022

Health Canada has granted approval for the usage of Moderna’s messenger RNA (mRNA) Covid-19 vaccine, Spikevax, in a 25µg two-dose regimen for active immunisation to prevent Covid-19 in children aged six months to five years. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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BioPharma Reporter

MAY 13, 2024

Circio, a biotech firm specializing in circular RNA-based gene therapy, has unveiled two posters showcasing in vivo proof-of-concept for its circVec platform at ASGCT 2024.

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Pharmaceutical Technology

AUGUST 2, 2022

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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MARCH 28, 2023

The gene-editing technology allows for precise, directed changes to genomic DNA. It edits genes by precisely cutting DNA, then allowing natural DNA repair processes to take over. The system comprises the Cas9 enzyme and a guide RNA. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

SEPTEMBER 1, 2022

These 'updated boosters' comprise the SARS-CoV-2 virus’ two messenger RNA (mRNA) components, one from the initial strain and the other one common between the BA.4 Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 5 lineage of the Omicron variant. In August, Moderna sought FDA EUA for the BA.4/BA.5

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STAT News

JANUARY 30, 2023

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. That said, a lot has changed in science and drug development since 1983. The human genome was sequenced in 2003.

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Pharmaceutical Technology

JULY 18, 2022

Moderna has received provisional registration from the Australian Therapeutic Goods Administration (TGA) for its messenger RNA (mRNA) Covid-19 vaccine, Spikevax, for kids aged six months to five years. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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BioSpace

OCTOBER 5, 2021

Voyager Therapeutics entered a deal with Pfizer that allows Pfizer to exercise options to license novel capsids created by Voyager’s RNA-driven TRACER screening technology.

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XTalks

JANUARY 3, 2024

Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to develop gene therapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). The total value of the deal could potentially reach $1.2 Sandrock, Jr., MD, PhD, CEO of Voyager.

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BioSpace

AUGUST 8, 2023

To build its ophthalmology portfolio, Japan’s Otsuka Pharmaceuticals has teamed with RNA editing biotech Shape Therapeutics to develop adeno-associated virus gene therapies for ocular diseases.

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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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Pharmaceutical Technology

JANUARY 12, 2023

The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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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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