XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

Gene Expression 112

Gene Expression Development RNA Drugs 112

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 52

Gene Editing DNA Genome Genomics 52

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 40

Gene Therapy Gene Gene Editing In-Vivo 40

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. CAMBRIDGE, Mass., 11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

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. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

RNA 52

RNA Protein DNA In-Vivo 52

The Pharma Data

OCTOBER 14, 2020

VBL has recently demonstrated ex-vivo activity of anti-MOSPD2 antibodies in patients with relapsing-remitting and progressive multiple sclerosis (MS), as well as in animal models of rheumatoid arthritis (RA), nonalcoholic steatohepatitis (NASH) and inflammatory bowel disease (IBD). 1. -- -->. -- [if lte IE 8]--> .

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing In-Vivo 52

The Pharma Data

DECEMBER 10, 2020

The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response. The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response.

Vaccine 52

Vaccine Vaccination Immune Response Protein 52

Roots Analysis

JANUARY 27, 2025

Protein therapeutics have completely revolutionized the healthcare segment, greatly influencing the study and treatment of human diseases by conferring precise targeting ability to therapeutic modalities. Considering their vast potential, close to 50% of the top selling drugs in 2023 were protein therapeutics.

Protein 40

Protein Reagent Marketing Gene 40

FDA Law Blog

DECEMBER 6, 2024

There is even a tip of the hat to alternative (non-animal) test methods, which have become powerful methods for the assessment of the potential for off-target toxicity and unintended genome editing. Section #1: FDA Interactions Given the wide range of sponsors (i.e., CBER will not commit to reviewing packages greater than 250 pages.

Gene Therapy 59

Gene Therapy Gene Production In-Vivo 59