HD Innovators Forum with Live Q&A Panel - Neubase, NeuExcell, Ophidian, PTC, and Triplet

(November 4, 2021) Dierich Stephan, PhD, from Neubase Therapeutics. Neubase Therapeutics is a differentiated platform for the discovery and development of novel genome-targeted drugs. Aims at targeting genes to increase proteins, decrease proteins, or edit protein function with selectivity and tolerability.
Russ Addis, Ph.D., from NeuExcell Therapeutics. NeuEXcell aims to regenerate lost GABAergic neurons using a novel neuro-regenerative gene therapy.
Douglas MacNeil, Ph.D., from Ophidian Biotechnology, leveraging a novel brain drug delivery platform treating neurodegenerative diseases with intrathecal / Intra-cranial delivery, transport across brain ventricles’ border, Trojan Horse (OCCT) allows systemic/IV administration, validated targets/monogenic disease silenced using siRNA/ASOs complexed to OCCT.
Irina Antonijevic, a neurologist and psychiatrist with a Ph.D. in neurophysiology, from Triplet Therapeutics. Somatic expansion in repeat expansion diseases and role of DNA damage response genes, clinical candidate antisense oligonucleotide (ASO) TTX-3360, Shield HD, and TTX-3360 Clinical Development plan.
Brian Pfister, Ph.D., from PTC Therapeutics. PTC Therapeutics is applying splicing technology to treat Huntington’s disease by targeting its root genetic cause. Their objective is to develop a disease-modifying HTT-lowering small module.