Quralis

QurAlis Corporation is a clinical-stage biotechnology company developing precision medicines for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and other neurodegenerative and neurological diseases with genetically validated targets. Its approach targets defined patient populations identified by disease-causing genetic mutations and clinical biomarkers, and centers on molecular drivers linked to TDP-43 pathology, including loss of STATHMIN-2 (STMN2), Kv7 channel dysfunction and mis-splicing of UNC13A.

The pipeline is led by QRL-201, a first-in-class candidate intended to restore STMN2 expression in ALS patients, evaluated in the ANQUR Phase 1 trial (NCT05633459), and QRL-101, a selective Kv7.2/7.3 ion channel opener addressed at hyperexcitability-induced disease progression in ALS and also being developed for epilepsy and pain (first-in-human Phase 1, NCT05667779). Additional programs extend the company's splicing expertise beyond neurodegeneration into indications such as Fragile X syndrome, and its stated areas of interest include epilepsy, pain, FTD and progressive supranuclear palsy. QRL-204, a program aimed at restoring UNC13A function in ALS and FTD, was exclusively licensed globally to Eli Lilly in 2024.

The company operates a proprietary antisense oligonucleotide splice-modulator platform branded FlexASO, which it reports shows higher potency and improved safety relative to standard ASOs, alongside human cellular disease models used for candidate development. Leadership includes founder and CEO Kasper Roet, CFO/COO Jason Brown, CSO Dan Elbaum, CTO Hagen Cramer, CMO Manoj Malhotra, Chief Business Officer Vikas Sharma and SVP of CMC Guzide Adhikari, with Anne C. Whitaker as board chair and directors representing Sanofi Ventures, DROIA Ventures, Mission BioCapital, Eli Lilly, EQT Life Sciences and Polaris Partners.

Founding story

QurAlis Corporation was founded on December 12, 2016 in Cambridge, Massachusetts by Kasper Roet, Kevin Eggan and Clifford Woolf together with Q-State Biosciences. The founders, neurodegenerative biologists associated with Harvard Medical School and Harvard University, built the company to translate discoveries about ALS disease drivers into precision therapies, including the 2014 finding by Eggan, Woolf, Wainger and Kiskinis that Kv7.2/7.3 dysfunction drives hyperexcitability-induced neurodegeneration and Eggan's 2019 discovery, published in Nature Neuroscience, that TDP-43 regulates STATHMIN-2 expression.

Business model

QurAlis develops proprietary therapeutic candidates in-house using its FlexASO antisense oligonucleotide splice-modulator platform and small-molecule programs, funding development through venture financing and monetizing selected assets through out-licensing to pharmaceutical partners, as with the exclusive global license of QRL-204 to Eli Lilly.

Pre-commercial; capital is raised from venture investors and value is realized through licensing of programs to larger pharmaceutical companies, such as the Eli Lilly agreements on Kv7.2/7.3 (2020) and QRL-204 (2024).

Traction

Two lead candidates have entered the clinic: QRL-101 began first-in-human dosing in a Phase 1 trial in December 2022/January 2023, progressed to multiple-ascending dose and ALS patient cohorts in 2024, and reported topline healthy-volunteer biomarker data in March 2025 and a target-engagement signal in ALS patients in December 2025. QRL-201 dosed its first patient in April 2023 in the ANQUR trial, advanced to dose range-finding in November 2024, and in February 2026 the company reported effects on disease progression and target engagement. The company also secured an exclusive global Lilly license for QRL-204 and an exclusive license on a novel Fragile X syndrome mechanism.

Latest developments

In November 2025 QurAlis appointed Manoj Malhotra, M.D., as Chief Medical Officer; in December 2025 it reported a signal of target engagement in ALS patients from the QRL-101 Phase 1 trial and was invited to present at the 44th Annual J.P. Morgan Healthcare Conference; in February 2026 it reported effects on disease progression and target engagement from the ANQUR trial of QRL-201; and in August 2026 it announced participation in September investor conferences.