Drug Discovery World

JANUARY 24, 2024

Fibrocor is on track to complete all IND-enabling work to support IND filing and initiation of clinical trials in 2024. A second challenge is that the available lab models mainly mimic the early stages of the disease, whereas patients available for clinical trials present in more advanced stages.

Research 59

Research Gene Expression Drugs Development 59

Delveinsight

JANUARY 18, 2021

Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father. The companies had to pause the trials of GTX-102 after the patients temporarily lost the ability to walk.

Marketing 52

Marketing Gene Drugs Protein 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. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

Gene Editing 52

Gene Editing Gene Engineer DNA 52