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.”

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Gene Editing Gene In-Vivo Gene Therapy 147

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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Gene Therapy Gene In-Vivo RNA 59

Worldwide Clinical Trials

APRIL 15, 2025

In vivo imaging using positron emission tomography (PET) provides spatial resolution of amyloid and tau deposition. Accurate diagnosis and effective disease monitoring remain challenging, particularly in the early stages, underscoring the critical need for robust biomarkers.

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BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

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Gene Editing Genetic Disease Gene In-Vivo 52

Scienmag

NOVEMBER 16, 2020

Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

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XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

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Gene Expression Development RNA Drugs 112

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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BioTech 365

JUNE 2, 2021

Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency Wave Life Sciences Announces Proof-of-Concept Preclinical Data for ADAR Editing Program in Alpha-1 Antitrypsin Deficiency First proof-of-concept in vivo data for RNA editing using endogenous … Continue reading →

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The Pharma Data

MARCH 8, 2021

This program included assays such as Big Blue and PIG-a designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo. The totality of the data from these studies indicates that molnupiravir is not mutagenic or genotoxic in in vivo mammalian systems. “We

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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. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

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The Pharma Data

DECEMBER 13, 2020

The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies.

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The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

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Gene Editing DNA Genome Genomics 52

XTalks

APRIL 22, 2022

Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry?

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Delveinsight

JANUARY 18, 2021

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. GeneTx and Ultragenyx are co-developing the therapy.

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The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

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Pharmaceutical Technology

FEBRUARY 14, 2023

Innovation S-curve for the pharmaceutical industry Mammalian expression vectors is a key innovation area in pharmaceutical Mammalian expression vectors are used to introduce a specific fragment of DNA into mammalian systems for RNA or protein expression. These systems include human and mouse cell lines, and even in vivo in live animals.

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XTalks

AUGUST 12, 2020

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).

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Delveinsight

FEBRUARY 25, 2021

Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs). Beam employs three approaches to deliver its genetic medicines to cells.

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Roots Analysis

AUGUST 31, 2023

It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E.

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The Pharma Data

JUNE 7, 2023

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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pharmaphorum

JANUARY 17, 2023

The intracellular barriers include endosomal escape, RNA sensors, and endonucleases. Ben Hargreaves speaks to Liberate Bio, a company that is hoping to address the delivery issues that could prevent mRNA, as well as siRNA and ASO therapeutics, from reaching their full potential.

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Roots Analysis

AUGUST 23, 2024

This adaptability was clear in coronavirus vaccine development using mRNA technology; after researchers had sequenced the virus’s RNA during January 2020, scientists were able create viable vaccine candidates within just months. This modern marvel wasn’t just used against COVID-19 but can be tailored now to fight any number of diseases.

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WCG Clinical

MAY 28, 2024

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.

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