The Cambridge, Massachusetts–headquartered biotech QurAlis kicked the year off by launching a Phase 1 clinical study of QRL-101 (QRA-244), a potentially novel selective Kv7.2/7.3 ion channel opener to treat hyperexcitability-induced disease progression in amyotrophic lateral sclerosis (ALS).
QRL-101 targets Kv7.2/7.3 potassium channels. Kv7 modulation can potentially lower spinal and cortical motor neuron excitability, potentially improving ALS patient survival.
QurAlis company is building a pipeline of antisense oligonucleotides (ASOs) and small molecule programs for ALS subtypes that constitute the majority of ALS cases. In addition to QRL-101, its pipeline includes QRL-201, QRL-203, QRL-204 and QRL-202.
The company’s QR43 Platform facilitates the discovery and development of novel therapies for TDP-43 pathologies, which are involved in some patients with ALS, frontotemporal degeneration and Alzheimer’s disease.
In an interview at the JP Morgan Healthcare Conference, QurAlis CEO Kasper Roet said he had long wanted to develop therapies for neurodegenerative diseases. “I wanted to do that ever since I got into neuroscience,” said Roet, who holds a Ph.D. in the subject.
There are about 40 genes that researchers have identified that predispose individuals to develop ALS.
Data analysis of the genetic underpinnings of ALS led Roet to conclude that ALS is becoming “a tractable problem.”
After deciding that he and fellow researchers could “actually do something for ALS patients,” Roet began comparing gene expression data sets from postmortem tissue, cell models and animal models.
As a self-described “big-data person,” Roet eventually concluded that animal models were ill-suited for ALS research.
In 2008, Kevin Eggan from Harvard University developed motor neurons from the skin cells of ALS patients.
Roet was inspired by the potential of using ALS patients’ cells to identify potential therapeutics.
Roet and Eggan, along with another stem cell pioneer, Clifford Woolf and Q-State Biosciences CEO Jonathan Fleming, founded QurAlis in 2016,
The company plans to have two proof-of-concept readouts and four programs in the clinic by 2025.
Filed Under: Neurological Disease