QurAlis
QurAlis is a clinical-stage biotechnology company developing precision antisense oligonucleotide therapies for ALS, frontotemporal dementia, Fragile X syndrome, and other neurodegenerative diseases using its proprietary FlexASO® Splice Modulator Platform.
- Company typePrivate
- Founded2016
- HeadquartersCambridge, United States
- Headcount51–100
- GTM typeB2B
- OfferingServices
What QurAlis does
QurAlis is a clinical-stage biotechnology company founded in December 2016 in Cambridge, Massachusetts, by Harvard neuroscientists Kasper Roet, Kevin Eggan, and Clifford Woolf alongside Q-State Biosciences. The company develops precision antisense oligonucleotide (ASO) medicines targeting genetically and clinically defined patient subpopulations within amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Fragile X syndrome, and other neurodegenerative and neurological diseases. QurAlis was the first to demonstrate that motor neurons derived from ALS patients via iPSC technology can be used to model disease and identify therapeutic targets, and operates a proprietary platform called FlexASO® Splice Modulator for generating splice-switching ASOs with improved potency, therapeutic index, and biodistribution. Its clinical pipeline comprises QRL-201 (first-in-class STATHMIN-2 restoring ASO for sporadic ALS, currently in Phase 2 ANQUR with up to 50% decrease in disease progression reported), QRL-101 (selective Kv7.2/7.3 ion channel opener for ALS hyperexcitability, Phase 1 with target engagement confirmed), and QRL-204 (UNC13A splice-correcting ASO for ALS/FTD, exclusively licensed to Eli Lilly).
The company is pre-revenue from a product sales perspective. Its revenue model is built on venture capital financing ($42M Series A in 2020, $88M Series B in 2023) supplemented by licensing and milestone payments from pharmaceutical partners — most notably the $45M upfront plus up to $577M in potential milestones from Eli Lilly for QRL-204. QurAlis has secured Clinical Trial Authorizations across Health Canada, the EU, and the UK, and operates a global clinical network spanning 11+ academic medical centers. Its go-to-market model is a traditional clinical-development-to-commercialization pathway: advance therapies through Phase 1/2/3 trials, secure regulatory approvals, then commercialize via specialty pharmacy and hospital distribution channels. The company recently doubled its physical footprint with a new 30,000 sq ft global headquarters and operates a Leiden, Netherlands European R&D and manufacturing facility, with leadership drawn from firms including Karuna Therapeutics (acquired by BMS for $14B), AbbVie, Retrophin/Pfizer, and Wave Life Sciences.
QurAlis firmographics
Firmographics- Name
- QurAlis
- Legal name
- QurAlis Corporation
- Website
- https://quralis.com
- Company type
- Private
- Founded year
- 2016
- Operating status
- Operating
- Headcount range
- 51–100 employees
- Short description
- QurAlis is a clinical-stage biotechnology company developing precision antisense oligonucleotide therapies for ALS, frontotemporal dementia, Fragile X syndrome, and other neurodegenerative diseases using its proprietary FlexASO® Splice Modulator Platform.
- Ownership category
- akta.pro rank
QurAlis industry classification
Industry- Product category
- ALS and Neurodegenerative Disease Precision Medicine
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)
- SIC
- Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)
- akta.pro secondary industries
- RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ), RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC) (HLAAADAF)
Keywords
Where QurAlis is headquartered
LocationHeadquarters
- HQ city
- Cambridge
- HQ country
- United States
- HQ region
- North America
Offices3 records
Markets served
QurAlis business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure
Revenue model
- Biopharmaceutical Development and Commercialization: As a clinical-stage biotechnology company, QurAlis generates no current revenue. The company is funded through equity financing (Series A and Series B venture funding) and licensing/partnership agreements. Future revenue will be generated through the commercialization of proprietary therapeutic candidates upon regulatory approval, through direct sales or licensing arrangements.
- Licensing and Milestone Payments: QurAlis has entered into an exclusive worldwide license agreement with Eli Lilly for QRL-204 (UNC13A-targeting ASO), receiving an upfront payment of $45 million plus equity investment, and is eligible for future milestone payments of up to $577 million and tiered royalties on net sales.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels6 records
QurAlis product offering
Product offeringCore offering
QurAlis is a clinical-stage biotechnology company developing precision antisense oligonucleotide (ASO) medicines that target defined genetic and molecular drivers of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases. Its wholly owned lead candidates are QRL-201 (first-in-class STATHMIN-2 restoring ASO for sporadic ALS, in Phase 1/2 ANQUR) and QRL-101 (selective Kv7.2/7.3 ion channel opener for ALS and epilepsy, in Phase 1). Its UNC13A asset QRL-204 has been out-licensed to Eli Lilly. All candidates are generated through the proprietary FlexASO® Splice Modulator Platform, which QurAlis also leverages for collaborations such as UMass Chan's Fragile X syndrome program.
Product overview
QurAlis is a clinical-stage biotechnology company and leader in developing precision therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The company's portfolio includes therapeutic candidates QRL-201 (first-in-class STATHMIN-2 restoring ASO for sporadic ALS) and QRL-101 (best-in-class Kv7.2/7.3 ion channel opener for ALS and epilepsy), both in clinical development. QRL-204 (UNC13A-restoring therapy) has been licensed to Eli Lilly. The FlexASO® proprietary platform enables development of splice-switching antisense oligonucleotides with improved potency and therapeutic index. The company is also advancing programs in frontotemporal dementia (FTD), Fragile X syndrome, and other neurological conditions.
Differentiator
Problem solved
Functional benefit
Brands
- FlexASO®: Proprietary Anti-Sense Oligonucleotide Splice Modulator Platform developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution
Products and services
- QRL-201 First-in-class antisense oligonucleotide precision medicine that restores STATHMIN-2 (STMN2) expression in patients with sporadic ALS, intended as a disease-modifying therapy targeting TDP-43-related loss of STMN2 in motor neurons. It is being evaluated in the ANQUR Phase 1/2 clinical trial (NCT05633459) for ALS patients.
- QRL-101 Potentially best-in-class selective Kv7.2/7.3 ion channel opener, an antisense/small-molecule-acting precision therapy intended to treat hyperexcitability-induced disease progression in ALS and is also being explored in epilepsy and pain. The Phase 1 program in ALS patients reported target engagement signals and EEG spectral power changes in beta and gamma bands.
- QRL-204 Potentially first-in-class splice-switching antisense oligonucleotide designed to restore UNC13A function in ALS and FTD by correcting TDP-43-driven UNC13A pre-mRNA mis-splicing; out-licensed exclusively worldwide to Eli Lilly and Company for development and commercialization.
Quantifiable outcome
- Up to 50% decrease in disease progression in ALS patients treated with QRL-201 vs. placebo, measured by ALSFRS-R score, across 69 patients in Phase 1/2 ANQUR trial
- +6 more outcomes
Companies that use QurAlis
Customer profileNamed customers2 records
Segments2 records
Ideal customer profiles3 records
QurAlis technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
QurAlis partnerships and signals
Strategic signalPartnerships
Six partnerships are on record, tiered minor, flagship and core.
- Healthpeak PropertiesminorHealthpeak Properties, QurAlis' landlord at 35 Cambridgepark Drive, developed and built out QurAlis' 30,000 square foot global headquarters facility including lab and office space. Healthpeak is a life science real estate company providing Class A lab space in Cambridge's Alewife Biotech Park.
- Eli Lilly and CompanyflagshipQurAlis granted Eli Lilly an exclusive worldwide license to develop and commercialize QRL-204 and other UNC13A-targeting ASO compounds for ALS, FTD, and other neurodegenerative diseases. QurAlis received $45 million upfront plus equity investment and is eligible for up to $577 million in future milestone payments plus tiered royalties on net sales. The agreement includes an R&D collaboration to identify and develop additional UNC13A-targeting candidates using QurAlis' FlexASO Platform. UNC13A mis-splicing occurs in up to 63% of ALS patients and up to one-third of FTD cases.
- UMass Chan Medical SchoolcoreQurAlis entered into an exclusive license agreement with UMass Chan Medical School on a novel RNA-targeted mechanism to restore functional protein for Fragile X syndrome (FXS). The collaboration with Dr. Joel Richter's lab confirmed FMR1-217 as a target for up to 80% of FXS patients, leveraging QurAlis' FlexASO platform for splice correction. Preliminary data suggest feasibility of a biomarker to detect mis-splicing of FMR1 in FXS patients.
- Quiver BioscienceminorQurAlis and Quiver Bioscience entered into a research collaboration to advance a novel gene-targeted therapeutic approach for Fragile X syndrome. The collaboration combines Quiver's Genomic Positioning System (GPS) drug discovery platform with QurAlis' ASO development expertise to build a data package for advancing FXS therapeutics.
- Patient Organizations (ALS Association, Project ALS, Target ALS, NEALS, I AM ALS, ALS Foundation, and others)minorQurAlis maintains partnerships with multiple ALS patient organizations to support clinical trial recruitment, patient engagement, and awareness. These organizations include ALS Association, ALS Foundation, Project ALS, Target ALS, NEALS, I AM ALS, and others. These partnerships facilitate patient-centered drug development and community outreach.
- Eli Lilly and Company (Kv7.2/7.3 Deal)coreQurAlis signed a deal with Eli Lilly in 2020 focused on Kv7.2/7.3 dysfunction in ALS, which built on QurAlis' foundational science showing that Kv7.2/7.3 dysfunction drives toxic hyperexcitability-induced neurodegeneration in ALS. This partnership contributed to the development of QRL-101.
Scale indicators8 records
Recent moves8 records
Expansion highlights7 records
QurAlis competitors and assessment
Company assessmentDirect peers
- Ionis Pharmaceuticals: Category-defining antisense oligonucleotide therapeutics company and the most direct competitor to QurAlis. Ionis developed Spinraza (nusinersen) for SMA and tofersen for SOD1 ALS, and operates a broad CNS ASO pipeline that directly overlaps with QurAlis' FlexASO platform approach. Its proven track record in ASO development and CNS delivery is the benchmark QurAlis is measured against.
- Wave Life Sciences: Clinical-stage RNA therapeutics company with a stereopure ASO platform targeting neurological diseases including ALS and Huntington's disease. Wave's PRISM platform overlaps directly with QurAlis' FlexASO chemistry approach, and its CTO Hagen Cramer's prior leadership at Wave makes it a particularly close technology analog.
- Stoke Therapeutics: Clinical-stage biotechnology company developing antisense oligonucleotide medicines that increase protein expression via RNA splicing modulation for genetic diseases, including CNS disorders such as Dravet syndrome and ADOA. Direct platform analog to QurAlis' FlexASO splice-modulator approach in the rare neurological disease space.
- Denali Therapeutics: Clinical-stage biotechnology company developing therapeutics for neurodegenerative diseases including ALS, Parkinson's, and Hunter syndrome. Denali is one of the most advanced and well-capitalized private competitors in the neurodegenerative precision-medicine space, directly competing for partnerships and ALS market positioning.
Broad incumbents
- Biogen: Large biopharmaceutical company commercializing tofersen (with Ionis) for SOD1 ALS and the broader Spinraza SMA franchise. Biogen is the dominant commercial incumbent in ALS therapeutics and represents the standard-of-care competitor QurAlis would face at launch, with far greater commercial infrastructure and reimbursement leverage.
- Alnylam Pharmaceuticals: Established RNA interference (siRNA) therapeutics leader with multiple approved products and an active CNS pipeline (e.g., for Huntington's disease). Competes with QurAlis in the broader RNA therapeutics space for capital, partnerships, and physician attention, though Alnylam's siRNA modality differs from QurAlis' ASO approach.
Emerging players
- ProQR Therapeutics: Clinical-stage RNA editing company developing Axiomer® platform-based therapies for genetic diseases including CNS and ophthalmology indications. Overlaps with QurAlis in RNA-targeted therapeutic modalities and rare neurological disease focus, though using an editing rather than splice-modulating mechanism.
- Karuna Therapeutics: Neuroscience-focused clinical-stage biotech acquired by Bristol-Myers Squibb for $14 billion in 2024 for its schizophrenia therapy. Karuna is a directly relevant precedent for QurAlis' exit trajectory given the shared CFO background (Jason Brown) and the demonstrated large-pharma appetite for CNS-focused clinical-stage biotechs.
- Sage Therapeutics: Clinical-stage neuroscience company developing CNS therapies (Zulresso, Zurzuvae) with overlapping neuroscience drug development infrastructure and investor base. Comparable in clinical-stage neuroscience focus and indication-overlap (epilepsy is one of QurAlis' expansion areas).
- Arrowhead Pharmaceuticals: Clinical-stage RNA therapeutics company with multiple modalities including siRNA and ASO-like trigger designs targeting liver and CNS diseases. Comparable as a private/public RNA therapeutics competitor with active neurology pipeline programs and broad platform ambitions similar to QurAlis' FlexASO strategy.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
QurAlis social profiles
Digital presenceQurAlis financial estimates
Financial estimateRevenue estimate
Valuation estimate
QurAlis leadership team
Management profileNumber of profiles
Profiles11 records
QurAlis funding detail
Funding detailFunding overview
Funding rounds4 records
Investors18 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
QurAlis M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about QurAlis
What does QurAlis do?
QurAlis is a clinical-stage biotechnology company developing precision antisense oligonucleotide (ASO) medicines that target defined genetic and molecular drivers of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases. Its wholly owned lead candidates are QRL-201 (first-in-class STATHMIN-2 restoring ASO for sporadic ALS, in Phase 1/2 ANQUR) and QRL-101 (selective Kv7.2/7.3 ion channel opener for ALS and epilepsy, in Phase 1). Its UNC13A asset QRL-204 has been out-licensed to Eli Lilly. All candidates are generated through the proprietary FlexASO® Splice Modulator Platform, which QurAlis also leverages for collaborations such as UMass Chan's Fragile X syndrome program.
Is QurAlis a public or private company?
QurAlis is a private company. It is classified as venture growth investor backed and is currently operating.
When was QurAlis founded?
QurAlis was founded in 2016. It employs 51 to 100 people.
Where is QurAlis based?
QurAlis is headquartered in Cambridge, United States, in the North America region.
How does QurAlis make money?
Two revenue lines are on record. Biopharmaceutical Development and Commercialization is the primary driver. The others are licensing and Milestone Payments.
Who are QurAlis's main competitors?
Direct peers on record are Ionis Pharmaceuticals, Wave Life Sciences, Stoke Therapeutics and Denali Therapeutics. Broad incumbents are Biogen and Alnylam Pharmaceuticals. Emerging players are ProQR Therapeutics, Karuna Therapeutics, Sage Therapeutics and Arrowhead Pharmaceuticals.
Does QurAlis have an API?
No public API is recorded for QurAlis.
What industry is QurAlis in?
QurAlis's product category is ALS and Neurodegenerative Disease Precision Medicine. Its primary akta.pro industry code is HLAAADAC, Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown), with a secondary code of HLAAADAG, RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics). Its NAICS code is 541714 and its SIC code is 2834.