Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

Gene Editing 94

Gene Editing Genetic Disease Genetics Protein 94

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

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. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

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. It’s a great tool for controlling gene expression.”.

DNA 52

DNA Gene Gene Silencing Genome 52

XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. RNA, and its protein-generating form messenger RNA (mRNA) discovered in 1961 , has quickly transitioned from being an obscure, finicky molecule that is difficult to work with, to becoming a significant cornerstone of therapeutic innovation in pharma and biotech.

Life Science 98

Life Science RNA Vaccination Vaccine 98