Pharmaceutical Technology

MAY 19, 2023

The financing will help to fast-track the development of other in vivo programming candidates into clinical trials. 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

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Pharmaceutical Technology

DECEMBER 11, 2022

In November, the companies announced plans for a Phase I clinical trial of the combined vaccine candidate in healthy adult subjects. 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.

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XTalks

JANUARY 8, 2024

Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. As Markham mentions, balancing the intricate demands of complex clinical trials without overburdening sites or patients is vital.

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Clinical Trials Clinical Trials Trials Gene Therapy 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. The KidCOVE trial was carried out at eight Canadian trial sites involving 414 children aged below five years.

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Vaccine Vaccination Gene Therapy Antibody 264

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. New ways of conducting clinical trials have also emerged. The human genome was sequenced in 2003.

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RNA Gene Therapy Drugs Gene 111

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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Gene Therapy Drugs RNA Clinical Trials 130

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

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Vaccination Vaccine Gene Therapy RNA 98

Pharmaceutical Technology

MAY 15, 2023

Rona Therapeutics has entered a partnership with Keymed Biosciences to discover and develop first-in-class siRNA [a double-stranded RNA molecule that is non-coding] therapeutics for glomerulonephritis, also known as severe kidney disease. The programme is expected to proceed towards clinical trials in the first half of 2024.

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Development Gene Therapy RNA Gene 130

Pharmaceutical Technology

NOVEMBER 30, 2022

in a project agreement from the US government for developing self-amplifying RNA (saRNA) vaccine technology against advanced and emergent viral threats. Additionally, the funds are intended to advance the vaccines to successful conclusion of Phase I clinical trials. By Cytiva Thematic.

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Pharmaceutical Technology

DECEMBER 14, 2022

An RNA editing therapy, WVE-006, could act on AATD’s liver and lung manifestations. On completion of the first-in-patient clinical trial by Wave, GSK will be in charge of the development and marketing works. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Life Science Development Gene Therapy Genetics 162

Pharmaceutical Technology

AUGUST 24, 2022

Mode rna has submitted an application to the US Food and Drug Administration (FDA) to obtain emergency use authorization (EUA) for mRNA-1273.222, its BA.4/BA.5 According to the findings, the trial of mRNA-1273.214 met all primary endpoints. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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XTalks

OCTOBER 26, 2021

Related: Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA. Givlaari reduces ALAS1 levels through RNA interference-mediated gene silencing, which targets ALAS1 mRNA. Clinical trial results have shown that Givlaari reduces severe AIP symptoms by 74 percent.

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Gene Silencing Gene Clinical Trials Clinical Trials 98

Delveinsight

JULY 24, 2021

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics.

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Gene Therapy Gene Gene Expression RNA 96

XTalks

JANUARY 21, 2025

In a clinical trial, participants demonstrated a 98% success rate after six months post implantation, with arteries widened successfully and no stent fractures observed. According to Geneoscopy , ColoSense is the first non-invasive colorectal cancer screening test to provide a dynamic view of disease activity by using RNA biomarkers.

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XTalks

JUNE 4, 2024

In the ever-evolving landscape of pharmaceutical development, the complexity of early phase clinical trials is increasing. The company partners with small and large biopharma and medical device and diagnostic companies to optimize early clinical development through excellence in study design and execution.

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Delveinsight

SEPTEMBER 14, 2021

ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. Novocure is also evaluating the therapy in non-small cell lung cancer.

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RNA Clinical Trials Clinical Trials Drugs 67

BioSpace

JUNE 23, 2022

BridgeBio released early data from its Phase I/II CANaspire clinical trial, indicating that BBP-812 might be a promising gene therapy for the ultra-rare Canavan disease.

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Gene Therapy Clinical Trials Clinical Trials Gene 40

FDA Law Blog

JANUARY 12, 2025

This may also help to derisk parallel product development amongst multiple products for the same condition, where another product in development may gain traditional approval or another product already on the market under accelerated approval may confirm clinical benefit prior to completion of clinical trials to support accelerated approval (e.g.,

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Clinical Trials Clinical Trials Trials Drugs 64

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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Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

JUNE 28, 2022

Listen to case studies presenting on pre-clinical and clinical research in areas such as oncology and what can be learned for future clinical trials. By attending the conference attendees will have the opportunity to. Understand the regulatory and CMC environment surrounding oligonucleotide therapeutic development.

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Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

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Marketing Gene Drugs Protein 52

XTalks

AUGUST 30, 2022

The webinar also featured Kelsey Carter, MS, Clinical Trial Manager at Medpace who discussed the site, operational and regulatory challenges for neuroscience trials with direct CNS administration along with the strategies to mitigate these challenges. Figure 3 ). Next Steps for Targeted CNS Delivery.

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Trials Drug Delivery Drugs Gene 83

pharmaphorum

APRIL 28, 2022

But you have access to a wide range of platforms and you have access to actually a very strong, experienced install-base in doing clinical trials, launching brands or commercialising them.”. Conversely, when you are a big pharma company, you have the shoulders to weather some disappointments from a safety perspective.

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Pharma Companies Branding Development Marketing 59

XTalks

APRIL 1, 2025

XTALKS WEBINAR: Keys to Success in Clinical Trials: A Strategic Guide for Biotechs and Startups Live and On-Demand: Thursday, May 22, 2025 , at 11am EDT (5pm CEST / EU-Central) Register for this free webinar to learn how biotechs can navigate endpoint challenges in clinical trials and accelerate their path to drug approval.

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FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

MARCH 23, 2023

China’s National Medical Products Administration (NMPA) has granted emergency use authorisation (EUA) for CSPC Pharmaceutical Group’s messenger RNA (mRNA) vaccine, SYS6006, to treat Covid-19. In April, CSPC Pharmaceutical secured emergency clinical trial approval to conduct trials of the mRNA vaccine. in clinical trials.

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XTalks

NOVEMBER 30, 2023

Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. Both trials concluded with maintained virologic suppression, and no clinically significant change from baseline in CD4+ cell counts was observed.

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FDA Approval Gene Therapy Clinical Trials Gene 111

Pharmaceutical Technology

FEBRUARY 1, 2023

At the time of Synagis’s approval, it was believed that continuous improvements in monoclonal antibody therapy and RSV drug development would follow, says Dr. Janet Englund, professor of Paediatric Infectious diseases at the University of Washington. However, subsequent RSV drugs, like Medimmune’s motavizumab, failed in clinical trials.

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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.

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Gene Therapy Gene Research Genetic Engineering 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Clinical Development Gene Therapy Development Gene 52

pharmaphorum

OCTOBER 12, 2022

The payload, Del Bourgo tell us, is the therapeutic DNA or RNA sequence that will cure or fix damaged cells. Inactive viruses often act as the viral vector, as they’re able to deliver genetic therapies efficiently. and France to accelerate the development of genomic therapies. So far, we’ve seen promising results.”.

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Genome Genomics Medicine Development 105

Roots Analysis

AUGUST 31, 2023

Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

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Pharmaceutical Technology

APRIL 27, 2023

GlobalData pharmaceutical analysts project that RNA-based gene therapies for oncology will grow from zero in 2022 to $4.6 There are currently 885 clinical trials ongoing or completed for prophylactic mRNA vaccines for infectious diseases and double the number of Phase I trials in 2022 compared to 2021.

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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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Vaccine Vaccination Gene Therapy Antibody 246

The Pharma Data

JANUARY 10, 2021

The new year began with a fairly low level of clinical trial news. Arcturus Therapeutics got the FDA go-ahead for its Phase II trial of its COVID-19 vaccine candidate ARCT-021. The trial will enroll 600 participants, with 450 receiving ARCT-021 and 150 receiving placebo. FBX-101 is a first-in-human AAV gene therapy.

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Gene Therapy Trials Gene Clinical Trials 52

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

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Delveinsight

AUGUST 5, 2021

The acquisition is the recent sign that large pharmaceutical companies view messenger RNA, which BioNTech and Moderna utilized to develop the COVID-19 vaccines now cleared for use in dozens of countries, as a crucial drug-making platform. Fibroblasts are part of the platform fueling cell-based gene therapy biotech Castle Creek.

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Vaccine Vaccination Gene Therapy Development 98

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies.

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Engineer Genetics Clinical Research Research 52