Broad Institute

AUGUST 24, 2022

The team then conducted analyses that triangulated treatment response, chromosomal deletion or gain, and concordant decreases or increases in mRNA and protein expression. Of the hundreds of genes deleted in this location, the DNA ligase gene LIG1 was one of the mostly consistently suppressed genes at both the mRNA and protein level.

DNA 52

DNA Protein Expression Treatment Protein Production 52

The Pharma Data

DECEMBER 3, 2020

Clinical Trials. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological Na V 1.1 December 6, 2020 12:00 PM – 1:30 PM; Track: 7.

Clinical Trials 52

Clinical Trials Protein Expression Pharmacokinetics Trials 52

The Pharma Data

JANUARY 13, 2021

However, before the Phase III program may begin, the FDA requested a meeting with Theratechnologies to discuss questions and comments received on certain aspects of the proposed trial design, to ensure alignment with the agency’s expectations with NASH trials. A second liver biopsy will be performed after 18 months.

Licensing 52

Licensing Licensing Clinical Trials Vaccine 52