The voltage-gated sodium channel family is composed of nine pore-forming members, which are named Nav1.1 to Nav1.9 and encoded by genes referred to as SCN1A to SCN11A

Dr. Kris Kahlig is the Head of Biology at Praxis Precision Medicines Inc. a clinical stage biopharmaceutical company translating genetic insights into the development of therapies for patients affected by CNS disorders. Kris presented at the Metrion Webinar series on “Using Dynamic Action Potential Clamp Modelling of Nav1.2 Variants to Support the Prediction of Clinical Phenotype in DEE”.

The webinar took place on Thursday 5th May 2022 at 16:00 BST.

We are delighted to present the fourth speaker in our “Ion Channels in Drug Discovery” webinar series – Dr. Greg Carr from the Lieber Institute for Brain Development. Greg’s presentation was entitled: “A role for hERG potassium channels in cognitive function”

The webinar took place on Thursday 3rd February 2022 at 16:00 BST.

We are delighted to present the third speaker in our webinar series – Professor David Sheppard from the University of Bristol. David’s presentation was focused on Cystic Fibrosis and his talk was “Cystic Fibrosis: Rescuing Faulty Channels with Targeted Therapies”.

The webinar took place on Tuesday 30th November 2021 at 16:00 BST.

Nonclinical safety pharmacology studies for siRNA follow a hybrid of the small molecule (SM) guidance and biologics guidance. The Oligonucleotide Safety Working Group (OSWG) has published a series of recommendation papers for oligonucleotides, including recommendations for safety assessment, because development of oligonucleotide-based therapeutics is not addressed by regulations. This is especially true regarding the hERG assay, which is a core assay in ICH S7B for SM. While OSWG states that a hERG study is not necessary for IND submission, all approved siRNAs have submitted hERG data which are necessary for requesting a TQT waiver.

We are delighted to present the second speaker in our webinar series – Professor Stephen Tucker from the Department of Physics at the University of Oxford. Stephen speaking on the topic of the TASK-1 K2P channel and his talk will be entitled “Defective X-gating caused by de novo mutations in the TASK-1 K2P channel (KCNK3) underlies a developmental disorder with sleep apnea”.

The webinar took place on Thursday 16th September 2021 at 16:00 BST.

One aim of the Comprehensive In Vitro Proarrhythmia Assay (CiPA) initiative is to improve drug safety testing in pre-clinical development by evaluating the proarrhythmic risk of a compound. Validation studies confirm that testing the effect of compounds on an increased number of human cardiac ion channel currents including INa (NaV1.5 peak and late current) as well as IKr (hERG) leads to improved prediction of their clinical risk.

To meet the FDA’s CiPA requirements for improved in silico action potential modelling and arrhythmia prediction, we have successfully created and implemented the challenging Milnes hERG cardiac safety assay

Rapidly activating and desensitising ligand-gated ion channel receptors can provide a technical challenge on automated patch clamp electrophysiology platforms. This can affect their biophysics, pharmacology and assay reliability. We present data on an optimised and validated acid-activated receptor assay on the QPatch that is stable enough for drug discovery screening.

As an alternative to standard pharmacological procedures for NaV1.5(Late) assays, we present a more reliable and accurate NaV1.5(Late) assay on QPatch that removes the requirement for activators like veratridine and ATX-II and delivers improved cardiac safety screening reliability and cost.