Pharmaceutical Technology

JUNE 1, 2023

A new intracellular drug delivery centre will be established in the UK to support potential ribonucleic acid (RNA) vaccines and therapeutics , as well as the development of innovative drug delivery technologies. It will focus on studying and developing new lipid nanoparticle (LNP) formulations for the delivery of RNA medicine.

Drug Delivery 242

Drug Delivery RNA Development Drugs 242

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.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

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

RNA 173

RNA Gene Therapy Gene Clinical Trials 173

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.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

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.

Gene Therapy 240

Gene Therapy RNA Gene Development 240

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.

Gene Therapy 99

Gene Therapy Gene Development RNA 99

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.

Genetics 328

Genetics Medicine Development Gene Therapy 328

Pharmaceutical Technology

AUGUST 15, 2024

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

Gene Therapy 130

Gene Therapy RNA Gene Clinical Trials 130

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.

RNA 130

RNA Medicine Gene Therapy Gene 130

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.

Development 241

Development RNA Gene Therapy Genetics 241

BioSpace

SEPTEMBER 22, 2020

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

RNA 96

RNA Gene Therapy Gene Protein 96

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.

Drugs 246

Drugs Gene Therapy Gene RNA 246

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.

RNA 147

RNA Vaccine Vaccination In-Vivo 147

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.

RNA 130

RNA Development Genome Genomics 130

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.

Gene Therapy 62

Gene Therapy RNA Gene 62

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.

Gene 110

Gene Gene Therapy Development RNA 110

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.

Vaccine 245

Vaccine Vaccination Development RNA 245

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.

RNA 261

RNA Gene Therapy Gene 261

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.

Vaccine 289

Vaccine Vaccination Gene Therapy Gene 289

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.

In-Vivo 246

In-Vivo Gene Therapy Gene RNA 246

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.

Gene Editing 235

Gene Editing DNA Gene Gene Therapy 235

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.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

BioPharma Reporter

OCTOBER 6, 2022

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

RNA 105

RNA Gene Therapy Gene Development 105

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.

Vaccine 289

Vaccine Vaccination Gene Therapy RNA 289

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.

Gene Therapy 59

Gene Therapy Gene In-Vivo RNA 59

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.

Vaccine 105

Vaccine Vaccination Gene Therapy RNA 105

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.

Vaccine 264

Vaccine Vaccination Gene Therapy Antibody 264

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.

Engineer 262

Engineer Vaccine Vaccination Clinical Trials 262

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

Vaccination 260

Vaccination Vaccine Gene Therapy RNA 260

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.

RNA 111

RNA Gene Therapy Drugs Gene 111

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.

Gene Therapy 52

Gene Therapy Gene Licensing Licensing 52

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.

RNA 98

RNA Manufacturing Gene Therapy Gene 98

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.

Vaccination 246

Vaccination Vaccine Gene Therapy Antibody 246

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.

Gene Therapy 78

Gene Therapy RNA Gene Licensing 78

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.

Gene Therapy 52

Gene Therapy Gene Development RNA 52

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027.

Gene Therapy 52

Gene Therapy Gene Research Genetic Engineering 52

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).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98