Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials.

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In-Vivo Gene Therapy Gene RNA 246

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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Vaccine Vaccination 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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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. 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 Vaccine Vaccination Clinical Trials 262

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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Vaccination Vaccine Gene Therapy Antibody 246

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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Vaccine Vaccination Gene Therapy RNA 105

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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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. 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

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

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

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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Gene Clinical Trials Clinical Trials Gene Therapy 204

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

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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Vaccine Vaccination 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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Vaccine Vaccination Development Gene Therapy 147

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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Vaccine Vaccination Gene Therapy Clinical Trials 130

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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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

Delveinsight

SEPTEMBER 23, 2021

858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. have helped prepare more than 20 drug candidates for clinical trials. Jeffrey Stafford, Ph.D., Stafford and co.

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RNA Gene Therapy Gene Protein 61

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 Gene Clinical Trials 111

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

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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Vaccination Vaccine Gene Therapy Development 98

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

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

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

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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Vaccine Vaccination Antibody Development 162

Roots Analysis

MAY 21, 2024

Several LNP-based therapies are currently being evaluated in the clinical trials; majority of these are conducted for treating breast cancer, ovarian cancer and lung cancer. In addition, these can be modified with ligands in order to improve the specificity and selectivity of the gene therapy and reduce off-target effects.

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Drug Delivery Drugs Gene Therapy Bioavailability 40

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

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