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.

Gene Editing 89

Gene Editing Gene DNA Engineer 89

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?

Life Science 98

Life Science RNA Vaccination Vaccine 98

Worldwide Clinical Trials

MAY 30, 2024

Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. Cambridge is a focal point for many pharmaceutical companies working in this therapeutic area.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

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 company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases.

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RNA Vaccination Vaccine Pharma Companies 98

pharmaphorum

JUNE 28, 2021

So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 90

In-Vivo Gene Editing Trials Drugs 90

Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality.

Research 52

Research Drugs Protein RNA 52

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. Peer went onto say that he believes that, in the near future, there will be many personalized treatments based on genetic messengers. Photo courtesy of Science Advances. ” Source link.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

SEPTEMBER 21, 2020

A study from the University of California San Francisco (UCSF) has revealed that women with Alzheimer’s do better than men with the disease due to a genetic advantage conferred by their extra X chromosome. The gene, called KDM6A , is a histone demethylase that is believed to function as a tumor suppressor. KDM6A Variant.

Gene 89

Gene Protein Gene Expression Scientist 89

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

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.

Life Science 111

Life Science Vaccination Vaccine Gene 111

Drug Discovery World

FEBRUARY 22, 2024

Indeed, we have shown that the causative mutations of some leukodystrophies affect the subunits of an important cellular enzyme, RNA polymerase III, preventing its normal assembly – it turned out that Riluzole can counteract this assembly defect,” says Maxime Pinard, the researcher responsible for the project in Coulombe’s lab. ‘’For

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59