pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

XTalks

NOVEMBER 30, 2023

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. This candidate, derived from a person’s own genetically modified cells, aims to restore the Gag-specific CD4+ T cell response in those receiving antiretroviral therapy.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics.

Gene 72

Gene Immune Response Manufacturing Gene Therapy 72

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

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

Gene Therapy 94

Gene Therapy Gene Gene Editing DNA 94

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 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

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. billion in funding. Cambridge is a focal point for many pharmaceutical companies working in this therapeutic area.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. A genetic treatment for epilepsy Most treatments for epilepsy aim to treat seizures, which is the main symptom of this condition, says Ferraro.

Research 264

Research Gene Gene Therapy Genome 264

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

Gene Editing 105

Gene Editing Gene Gene Therapy Clinical Trials 105

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

Drug Discovery World

AUGUST 2, 2022

CureHeart is using gene therapy technologies to edit or silence the faulty genes that cause inherited heart muscle diseases. . Also known as genetic cardiomyopathies, they often go undiagnosed and are a major cause of heart transplants due to how current treatments do not prevent the condition from progressing. .

Gene Editing 52

Gene Editing Gene Research Genetics 52

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

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. While type II (Cas9) is mainly used for gene-editing applications, CRISPR detecting tools primarily rely on type V (Cas12) and type VI (Cas13) collateral activity.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

MAY 30, 2023

At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality. The concept of harnessing the ubiquitin-proteosome system to ‘destroy’ or degrade disease causing proteins has been established for some time.

Research 52

Research Drugs Protein RNA 52

Drug Discovery World

FEBRUARY 22, 2024

Leukodystrophies are rare and almost exclusively genetic diseases characterised by a process of demyelination (damage to the myelin sheath) of the central and peripheral nervous system. They focused on miRNAs, which regulate multiple target proteins controlling the cellular processes in the amygdala.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59