Anu Bhattacharyya

Company: PTC Therapeutics

Job title: Vice President, Splicing Drug Development

Seminars:

Roundtable Discussion: Evaluating Regulators’ Views on Safety & Toxicity Testing to Streamline Preclinical Testing & Fast-Track RNA-Targeting Drug Approval 9:30 am

What are regulators' thoughts on using animal models to showcase off-target effects and toxicity given their limited RNA conservation? What safety and toxicity tests are required to gain FDA approval for advancing into human clinical trials? Highlight specifications which are required to be met by RNA-targeting drugs to ensure regulatory approvalRead more

day: Focus Day

PTC518 Mediated Splice-Induced HTT Lowering: Pathway from Concept to Patients 9:30 am

Uncovering how animal models demonstrated that reducing mutant huntingtin protein (mHTT) levels could alleviate Huntington’s disease (HD)-like symptoms Reviewing the discovery and development of PTC518, a small molecule splicing modifier that reduces the production of the mHTT protein in cells and animals Highlighting PTC518’s clinical data to demonstrate proof of mechanism and dose-dependent lowering of…Read more

day: Day Two