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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Vaccination Vaccine Gene Therapy Gene 289

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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Vaccination Vaccine 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. Following the demonstration at Samsung, the clinical trial of GreenLight’s Covid-19 booster vaccine is anticipated to commence this year.

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Engineer Vaccination Vaccine Clinical Trials 262

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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Vaccination Vaccine Gene Therapy RNA 105

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

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. It’s important to make sure that with this increase in clinical trial complexity, we don’t make our trials overly burdensome to sites or patients,” says Markham.

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Clinical Trials Clinical Trials Trials Gene Therapy 105

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. Development of vaccines to Phase I trials under the five-year $59m prototype project comprises additional $28.4m in milestone payments. By Cytiva Thematic.

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Vaccination Vaccine Development Gene Therapy 147

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. At present, a Phase II/III trial of mRNA-1273.222 is progressing. 1 subvariant.

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Vaccination Vaccine Gene Therapy Antibody 130

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. By Cytiva Thematic.

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Life Science Development Gene Therapy Genetics 162

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. In a trial with 100 patients , the device demonstrated no major complications, an average bleeding stop time of 2 minutes, with patients walking within approximately 2.5

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FDA Approval Manufacturing Development RNA 105

XTalks

OCTOBER 26, 2021

The Phase III trial results show that Givlaari offers sustainable benefit to AIP patients and confirm its favorable safety profile. Related: Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA. This is a huge development for patients.”.

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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

JUNE 4, 2024

In the ever-evolving landscape of pharmaceutical development, the complexity of early phase clinical trials is increasing. Cohen speaks about the significant increase in complexity in the early clinical space, discussing factors including increasing development costs and increasing complexity of molecules and resulting trial designs.

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Clinical Development Development Clinical Trials Clinical Trials 110

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. Ixaka remains blinded to the data until the execution of the trial.

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RNA Clinical Trials Clinical Trials Drugs 67

XTalks

AUGUST 30, 2022

Clinical research experts from the CRO Medpace shared insights about the operational and regulatory considerations for neuroscience trials with direct CNS administration. Another advantage of intrathecal delivery is that it may allow therapies to gain access to the spinal space which may not have systemic access to the CNS.

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Trials Drug Delivery Drugs Gene 83

pharmaphorum

MARCH 9, 2022

The XTEND-1 trial also showed that BIVV001 was better at reducing the annualised rate of bleeding episodes than patients’ previous therapies, according to Sanofi and Sobi, which said they will now talk to regulators to chart a path forward to approval.

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Protein Gene Therapy Sales Regulation 98

FDA Law Blog

JANUARY 12, 2025

Despite including guidance on confirmatory trials and FDAs authority to require them, the Agency decided to publish an additional guidance focused solely on how it is interpreting the authority to require that such trials be underway prior to accelerated approval or within a specified time period after the date of accelerated approval.

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Clinical Trials Clinical Trials Trials Drugs 64

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

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

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression). References.

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Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

JULY 11, 2022

Sanofi R&D in its core haemophilia category is advancing on three fronts – engineered proteins, RNA interference drugs and gene therapies – and highlighted results in two of those at the International Society on Thrombosis and Haemostasis (ISTH) congress over the weekend.

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Engineer Gene Therapy Sales Drugs 52

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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RNA Development Engineer Clinical Trials 52

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

pharmaphorum

JULY 21, 2021

It is a small interfering RNA (siRNA) therapy that works by inhibiting PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month. Its innovative medicines division climbed 15% to $10.6 Leukaemia dug asciminab filed.

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Sales Gene Therapy RNA Production 52

pharmaphorum

APRIL 28, 2022

Although they have, by definition, small addressable markets, the treatments cost just as many resources to develop as therapies for more common diseases – sometimes more, because of the added difficulty of finding sufficiently large patient populations for trials and finding disease experts to run them.

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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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Vaccination Vaccine Gene Therapy Clinical Trials 130

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 Clinical Trials 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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Vaccination Vaccine Antibody Development 162

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% An IRB tends to get the most attention since all human trials need IRB approval.

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Gene Therapy Gene Research Genetic Engineering 52

pharmaphorum

AUGUST 22, 2022

” For example, Torrance recently met with an expert from the Broad Institute who has been looking into gene therapies and says gene therapy can completely change how drugs are built. Exscientia currently has three candidates in phase one trials. Often it’ll be quite serendipitous.”

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Gene Therapy Antibody Gene Medicine 102

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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Gene Gene Therapy Gene Expression Regulation 40