Genomics, In-Vivo and RNA - Clinical Research Informer

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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo

In-Vivo Gene Editing Trials Drugs

Moderna partners with Life Edit for mRNA gene editing therapies

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. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

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Bridging Innovation & Patient Care: The Growing Role of AI

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Trending Sources

Shining a light on the role of the genome’s ‘dark matter’ in cancer development

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. Genome Med 12, 89 (2020). Harmston, N., Glaser, T.L.

In-Vivo

In-Vivo RNA DNA Genome

Webinars

Bridging Innovation & Patient Care: The Growing Role of AI

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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

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.

Gene Expression

Gene Expression Development RNA Drugs

Verve starts trials of cholesterol drug in test of base editing technique

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.

Gene Editing

Gene Editing Trials Drugs In-Vivo

EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– EdiGene, Inc. as Head of the US Subsidiary, and Kehua Fan, M.D.,

Business Development

Business Development Development Gene Editing In-Vivo

CRISPR Cas-9 System Doubles Life Expectancy in Mice with Aggressive Cancers

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.

Gene Editing

Gene Editing DNA Genome Genomics

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

DECEMBER 21, 2020

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

Genetics

Genetics Medicine Gene Therapy Marketing

ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates

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.

Vaccine

Vaccine Vaccination Immune Response Protein

Leading innovators in mammalian expression vectors for the pharmaceutical industry

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.

In-Vivo

In-Vivo DNA Antibody Gene

What the Glycome Can Tell Us About Persistent HIV Infection

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).

RNA

RNA Protein DNA In-Vivo

Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer

Delveinsight

FEBRUARY 25, 2021

Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The GuideTx technology screens for lipid nanoparticles inside the body that can be used to deliver RNA to cells throughout the body.

DNA

DNA In-Vivo Genetics Medicine

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

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.

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

Moderna partners with Life Edit for mRNA gene editing therapies

Webinars

Trending Sources

Shining a light on the role of the genome’s ‘dark matter’ in cancer development

Webinars

Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

Verve starts trials of cholesterol drug in test of base editing technique

EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

CRISPR Cas-9 System Doubles Life Expectancy in Mice with Aggressive Cancers

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates

Leading innovators in mammalian expression vectors for the pharmaceutical industry

What the Glycome Can Tell Us About Persistent HIV Infection

Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

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