The startup develops precision therapeutics and diagnostic tools targeting genetically validated alterations in amyotrophic lateral sclerosis and other neurodegenerative diseases. Its proprietary platforms enable the design of drugs that can halt disease progression and improve patient outcomes through direct intervention on genetic factors.
Funding
$138.5M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
ELFounders
Product
Problem
Amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases lack effective treatments that address the underlying genetic causes and disease mechanisms. Many current treatments only provide symptomatic relief without modifying the course of the disease. A significant unmet need exists for precision therapies that target specific components of disease pathology and defined patient populations.
Solution
QurAlis is developing precision medicines and diagnostic tools that target genetically validated alterations in ALS, FTD, and other neurodegenerative and neurological diseases. The company's FlexASO® platform generates splice-switching antisense oligonucleotides (ASOs) with improved potency, increased therapeutic index, improved biodistribution, and reduced off-target activity. QurAlis is advancing a pipeline of therapeutic candidates, including QRL-201, which aims to restore STATHMIN-2 (STMN2) expression in ALS patients, and QRL-101, a selective Kv7.2/7.3 ion channel opener for hyperexcitability-induced disease progression in ALS, epilepsy, and pain. These therapies aim to modify disease progression and improve patient outcomes by directly intervening on genetic factors and disease drivers.
Target Audience
The primary target audience includes patients with ALS, FTD, epilepsy, and other neurodegenerative diseases, as well as researchers and clinicians focused on developing and delivering precision medicine therapies for these conditions.
Features
- FlexASO® platform for generating splice-switching ASOs with improved potency and reduced off-target activity
- QRL-201: A first-in-class molecule for the treatment of ALS that aims to restore STMN2 expression
- QRL-101: A selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, epilepsy, and pain
- Therapeutic candidates targeting STATHMIN-2 (STMN2), Kv7, and UNC13A
- Clinical trials underway for QRL-201 (ANQUR trial) and QRL-101
- Focus on precision medicine approach, targeting specific components of disease pathology and defined patient populations based on genetic mutations and clinical biomarkers