AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.

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Genetic Disease Genetics Gene Editing Medicine 189

BioSpace

JUNE 12, 2022

Shape Therapeutics is evolving programmable RNA medicines that push the limits of current gene editing capabilities. BioSpace spoke with CSO David Huss.

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Gene Editing RNA Gene Medicine 71

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Gene Therapy Gene Gene Editing Development 243

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

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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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pharmaphorum

NOVEMBER 1, 2022

14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Date: 8 – 9 February 2023.

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BioPharma Reporter

JUNE 20, 2024

With Roche as a partner, Ascidian Therapeutics aims to treat conditions that currently cannot be addressed by existing gene editing technology

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XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

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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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pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

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XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

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Life Science RNA Vaccine Vaccination 98

pharmaphorum

JANUARY 10, 2022

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The biotech is focused on mRNA but has developed a self-amplifying mRNA (sa-RNA) platform in an attempt to develop the potential of the technology further.

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RNA Vaccine Vaccination Pharma Companies 111

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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Bacteria Gene Editing Research RNA 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells. Source link: [link]

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The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Photo courtesy of Science Advances.

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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. SR One Capital Management and OrbiMed Advisors co-led Series B. Simeon George, M.D.,

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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. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV. percent in the placebo group.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenetic Editing with CRISPR.

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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. greater safety, transient and reversible effect on gene expression).

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The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO

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Delveinsight

JANUARY 14, 2021

Last year, the company disclosed its gene-writing service, which combined various gene editing, manufacturing, and synthesizing technologies to provide more tailored therapeutic instructions to genetic code. Verve Therapeutics has an aim to bring one-and-done gene editing treatments to heart disease.

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Gene Editing Gene Genetics DNA 40

The Pharma Data

MARCH 7, 2022

Cas9 is the protein used in the gene editing process called CRISPR. Cas9 binds and uses RNA as an address-tag. The system allows scientists, by synthesizing a specific “address-tag” RNA, to bring Cas9 to a precise location in genome and therefore cut and splice genes at specific sites. .”

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Protein Gene Genomics Genome 52

The Pharma Data

DECEMBER 5, 2020

The first of the new therapies, developed at Harvard Medical School by a team led by Dr. David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. The CRISPR gene editing method won the 2020 Nobel Prize in Chemistry, and uses chemicals to open up and directly edit DNA sequences, Hsu said.

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Gene Therapy Gene Gene Editing Genetics 52

Bioengineer

JULY 23, 2021

“By applying high-resolution imaging, advanced cell and molecular readouts and RNA sequencing methods leveraging the OTME-Chip, we discovered the actual genetic signaling pathways behind the blood cell triggered metastasis of ovarian cancer and a new drug strategy to stop this process.” ” ###.

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Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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DNA In-Vivo Genetics Medicine 52

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenome Editing with CRISPR.

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DNA Gene Gene Silencing Genomics 52

XTalks

SEPTEMBER 4, 2024

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

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Worldwide Clinical Trials

MAY 30, 2024

An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient. The targeted ASOs aim to downregulate the expression of gene mutations that are associated with gain-of-function toxicity that leads to motor neuron loss in some ALS cases.

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XTalks

AUGUST 2, 2023

2) Veklury (remdesivir) Veklury, approved by the FDA in 2020, is a SARS-CoV-2 nucleotide analog RNA polymerase inhibitor designed for the treatment of COVID-19. Additionally, Bayer has formed a partnership with Mammoth Biosciences to broaden its product portfolio with innovative gene-editing technology. billion in 2022.

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Sales Manufacturing FDA Approval Life Science 98

XTalks

SEPTEMBER 21, 2020

A female’s second X chromosome is typically silenced (so that females don’t get an overdose of X chromosome genes) through X-linked inactivation, a process where the chromosome is coated with an outer layer of non-coding RNA. One of these doubly active genes is KDM6A.

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

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Pharmaceutical Technology

FEBRUARY 20, 2023

Gene therapy, while in its infancy, could be a gamechanger for cancer treatment and certain rare diseases and advancements in gene-editing technology relies on knowledge of viral factors. The opportunity here for our expanded CRO to advance personalised medical solutions is huge.

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XTalks

FEBRUARY 11, 2025

Gene therapy has shown promise in treating cancers that are particularly difficult to manage such as neuroblastoma and Wilms tumor using CAR-T cell therapy, CRISPR-Cas9 gene editing and RNA-based interventions to target genetic drivers of disease.

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