n-Lorem Foundation
n-Lorem Foundation is a 501(c)(3) nonprofit that designs and provides personalized experimental antisense oligonucleotide (ASO) medicines, free for life, to nano-rare disease patients (1-30 patients worldwide per mutation). Founded in 2020 by Ionis Pharmaceuticals founder Stanley Crooke, it has treated 50+ patients with 260+ cumulative doses.
- Company typePrivate
- Founded2020
- HeadquartersCalifornia City, United States
- Headcount1–10
- GTM typeB2C
- OfferingServices
What n-Lorem Foundation does
n-Lorem Foundation is a 501(c)(3) nonprofit founded in January 2020 by Dr. Stanley Crooke — the founder of Ionis Pharmaceuticals and creator of antisense oligonucleotide (ASO) technology — to develop and provide personalized experimental ASO medicines to nano-rare disease patients (defined as 1-30 patients worldwide sharing a single pathogenic mutation) for free, for life. The foundation is the only organization worldwide operating at scale on individualized ASOs, with 50+ patients treated, 260+ doses administered, 40+ INDs filed across five FDA divisions plus one CTA with Health Canada, and over 45 patient-years of cumulative safety experience as of April 2026.
The core product is a personalized experimental ASO medicine engineered per patient using the foundation's proprietary discovery platform: massive parallel screening of thousands of mRNA sites is coupled with machine-learning algorithms to optimize drug-like properties, and six distinct molecular mechanisms (RNase H1-mediated degradation, splicing modulation, nonsense-mediated decay inhibition, uORF targeting, polyadenylation redirection, and translation inhibition) are available to either up- or down-regulate protein expression. Organ-specific delivery expertise covers the central nervous system, liver, kidney, and eye via subcutaneous, intravitreal, and intrathecal routes. Quality and pre-clinical work is supported by Charles River Laboratories; manufacturing by Nitto Avecia, ChemGenes, Hongene and Cytiva; sequencing and diagnostics by Illumina, GeneDx, IDT, Children's Mercy Kansas City and (proposed) PacBio; and clinical operations by Parexel. The foundation reports >90% clinically significant benefit rates across evaluable patients with no ASO-related serious adverse events.
The business model is philanthropic rather than commercial. Revenue is generated from charitable donations and directed gifts from pharmaceutical and biotech partners (Biogen as founding donor, Alexion, Alnylam, Takeda, Ultragenyx, Servier, GondolaBio), foundations (Conrad Prebys, Solve FSHD, Wolverine, J. Wood Capital), federal and state grants (including NIH support via the Undiagnosed Diseases Network), and milestone/royalty arrangements from commercial partnerships. Average per-treatment cost is approximately $1.2 million, largely subsidized through in-kind manufacturing and reagent donations. Go-to-market is community-led: research physicians at tertiary care centers submit applications on behalf of genetically confirmed patients via the foundation's Application for Treatment Portal, and patients engage through the Patient Empowerment Program, an annual Nano-rare Patient Colloquium (hosted by Biogen), and the "Lessons in Antisense" podcast. The foundation is governed by a Board of Directors chaired by Crooke, employs 31 people, and operates from San Diego with a second laboratory in El Cajon.
n-Lorem Foundation firmographics
Firmographics- Name
- n-Lorem Foundation
- Legal name
- n-Lorem Foundation
- Website
- https://nlorem.org
- Company type
- Private
- Founded year
- 2020
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- n-Lorem Foundation is a 501(c)(3) nonprofit that designs and provides personalized experimental antisense oligonucleotide (ASO) medicines, free for life, to nano-rare disease patients (1-30 patients worldwide per mutation). Founded in 2020 by Ionis Pharmaceuticals founder Stanley Crooke, it has treated 50+ patients with 260+ cumulative doses.
- Ownership category
- akta.pro rank
n-Lorem Foundation industry classification
Industry- Product category
- Personalized Genomic Medicine
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)
- SIC
- Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)
- akta.pro secondary industries
- Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH)
Keywords
Where n-Lorem Foundation is headquartered
LocationHeadquarters
- HQ city
- California City
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
n-Lorem Foundation business model
Business model- GTM type
- B2C
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Supply Chain, Operations, Infrastructure, Marketing or Sales, Others
Revenue model
- Charitable Donations and Philanthropy: Primary funding source including donations from pharmaceutical companies (Biogen, Alexion, Alnylam, Takeda, Ultragenyx, Servier), biotechnology companies, foundations (Conrad Prebys Foundation, SolveFSHD, Wolverine Foundation), and individual donors. The organization received over $30 million in cash and significant in-kind donations within the first 18 months. In 2025, charitable contributions grew meaningfully with several major donors added.
- Federal and State Grants: Funding from government sources including NIH support through the Undiagnosed Diseases Network and other grant programs.
- Directed Donations: Support for specific gene or mutation research programs. For each directed donation, n-Lorem requests an additional donation to the general fund for treating all patients. Directed donors include industry partners funding specific ASO programs.
- Commercial Partnerships - Milestones and Royalties: Relationships with commercial partners generate funding through milestone payments and potential royalties. These transactions are subject to strict criteria ensuring consistency with n-Lorem's mission. In 2025, milestones were achieved from existing partnerships.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| Other | Others | Free treatment for all qualified nano-rare patients |
Go-to-market motion2 records
Distribution channels2 records
Marketing channels7 records
n-Lorem Foundation product offering
Product offeringCore offering
n-Lorem Foundation develops and provides personalized experimental antisense oligonucleotide (ASO) medicines, for free for life, to patients with nano-rare genetic diseases (1-30 patients worldwide with the same mutation). Each treatment is designed for an individual patient's unique single-gene mutation affecting the central nervous system, liver, kidney, or eye, leveraging 30+ years of ASO technology expertise developed at Ionis Pharmaceuticals.
Product overview
n-Lorem Foundation operates as a single, unified service offering centered on providing personalized experimental antisense oligonucleotide (ASO) medicines to nano-rare patients. The core offering is the ASO Technology Platform, which enables the discovery and development of individualized ASO medicines for patients with single-gene mutations affecting 1-30 patients worldwide. Supporting services include a Patient Application Portal for treatment evaluation, an annual Nano-Rare Patient Colloquium for community engagement, and a Patient Empowerment Program providing education and support resources. All treatments are provided at no cost to patients for life, representing a charitable nonprofit model rather than a commercial pharmaceutical product portfolio.
Differentiator
Problem solved
Functional benefit
Products and services
- Personalized Experimental ASO Medicines Individualized antisense oligonucleotide (ASO) medicines developed for each nano-rare patient with a confirmed single-gene mutation affecting the central nervous system, liver, kidney, or eye. Each ASO is a short, modified DNA strand designed to bind precise RNA target sites and modulate disease-causing protein expression via mechanisms such as RNase H1-mediated degradation, splicing modulation, nonsense-mediated decay inhibition, uORF targeting, polyadenylation redirection, or translation inhibition. Provided free for life to qualifying patients under Research INDs.
Quantifiable outcome
- Greater than 90% success rate with clinically significant benefit achieved in nearly all evaluable patients
- +5 more outcomes
Companies that use n-Lorem Foundation
Customer profileNamed customers3 records
Segments3 records
Ideal customer profiles3 records
n-Lorem Foundation technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature5 records
n-Lorem Foundation partnerships and signals
Strategic signalPartnerships
17 partnerships are on record, tiered core.
- ServiercoreMulti-target research collaboration to develop antisense oligonucleotide (ASO) therapeutic approaches for rare neurodevelopmental disorders. n-Lorem leverages its ASO technology platform to engineer preclinical candidates, which Servier advances into clinical development. Partnership addresses rare neurological diseases including refractory epilepsy, genetically-driven autism spectrum disorders, and leukodystrophies. Supports Servier's 2030 ambition for rare neurological diseases.
- Critical Path Institute (C-Path)coreFounding member of One to Millions initiative - a global public-private initiative to enable scalable development of advanced individualized therapies such as ASOs, gene therapies, and RNA-based treatments. Initiative creates standardized development pathways, integrates post-authorization evidence generation, and reduces unnecessary animal testing. Also includes Mila's Miracle Foundation and N=1 Collaborative.
- EspeRare FoundationcoreStrategic collaboration to expand access to n-Lorem's individualized ASO therapies in Europe. Initial focus on treating a small number of patients in Switzerland as proof-of-concept before expanding across the EU. Addresses regulatory, operational, and diagnostic barriers limiting access to personalized genetic medicines outside the United States. First treatments anticipated within the first year.
- Pacific Biosciences (PacBio)coreProposed strategic collaboration to leverage PacBio's long-read whole-genome sequencing technology for developing individualized ASO therapies for ultra-rare genetic diseases. PacBio would donate sequencing reagents and provide scientific expertise for long-read genome analysis to reduce sequential testing, accelerate target validation, and improve ASO design confidence. Remains subject to negotiation.
- AlnylamcorePartnership to expand technology access to RNAi therapeutics for patients with nano-rare diseases where an RNAi directed approach would be most useful. Alnylam's vision is to harness potential of RNAi therapeutics for limited treatment options.
- ParexelcoreClinical partner providing clinical operations, real-world data solutions, medical and regulatory expertise and innovative clinical research tools. One of world's leading global clinical research organizations. Helps manage the complex database and natural history studies for n-Lorem's patient programs.
- Charles River LaboratoriescoreProvides support for substantial amount of pre-clinical toxicity studies and data collection to support regulatory filing. Global provider of drug discovery and development services.
- Children's Mercy Kansas CitycoreLeading independent children's health organization providing genomic sequencing support. Dedicated to holistic care, translational research, breakthrough innovation, and educating next generation of caregivers.
- GeneDxcoreHelps understand the genetic cause of nano-rare mutations. Leader in delivering clinical genomic answers to patients, families and healthcare providers.
- IlluminacoreSupports nano-rare patients by sequencing patient genomes to identify and understand each patient's unique mutation. Goal is to apply innovative technologies to analysis of genetic variation and function.
- ChemGenes CorporationcoreProvides significant manufacturing support for n-Lorem and patients. Helps offset costs of developing personalized experimental ASO medicines.
- Nitto AveciacoreDonor and partner providing manufacturing support through contributions of active pharmaceutical ingredients (API) and drug product for individualized ASO medicines. Over 25 years of oligonucleotide innovation through development and production.
- Target ALScoreNon-profit organization envisioning world where everyone with ALS lives. Partnership supports ALS patient characterization and ASO development.
- Silence ALScoreInitiative aligning Columbia University and n-Lorem to support characterization of nano-rare ALS patients and discovery and development of personalized experimental ASO medicines for these patients. Founded by Dr. Neil Shneider.
- Wolverine FoundationcoreNon-profit advancing research and discovering novel therapeutic approaches for neurodevelopmental disease caused by genetic variations in MAPK8IP3 gene.
- Undiagnosed Diseases Network (UDN)coreNIH-funded research study bringing together clinical and research experts to solve most challenging medical mysteries using advanced technologies. Provides access to appropriately characterized patients and investigators.
- GondolaBiocoreClinical-stage biotechnology company dedicated to developing novel medicines for patients living with genetic diseases. Partner in discovering novel ASO medicines for patients not currently served by available treatment options.
Scale indicators10 records
Recent moves6 records
Expansion highlights6 records
n-Lorem Foundation competitors and assessment
Company assessmentDirect peers
- Mila's Miracle Foundation: Nonprofit foundation created to develop a personalized antisense oligonucleotide treatment for Mila Makovec, a child with Batten disease. Like n-Lorem, it pioneered the individualized ASO concept for a single patient and now co-leads the One to Millions initiative with n-Lorem. Directly comparable as the original N-of-1 ASO foundation.
- N=1 Collaborative: Organization focused on advancing individualized therapeutics for patients with rare genetic diseases. Co-founding member of the One to Millions initiative alongside n-Lorem and Mila's Miracle. Directly comparable mission and approach to enabling individualized medicine development.
- EspeRare Foundation: Swiss nonprofit foundation partnering with n-Lorem (announced January 2026) to expand individualized ASO therapies into Europe, initially Switzerland. Operates a comparable nonprofit model focused on developing treatments for ultra-rare diseases that lack commercial paths.
Broad incumbents
- Ionis Pharmaceuticals: The originator of ASO technology where founder Stanley Crooke led creation of the platform for 30+ years. Commercial ASO developer with a broad pipeline across rare and common diseases. Comparable because n-Lorem's technology platform is a direct spinoff of Ionis's ASO expertise and several senior staff are Ionis alumni.
- Biogen: Leading global biotechnology company that is n-Lorem's founding donor and host of the annual Nano-rare Patient Colloquium. Licensee of ASO technology from Ionis with multiple commercial ASO products (e.g., Spinraza, Tofersen). Comparable because Biogen supports n-Lorem's mission while also operating in the broader ASO and rare disease neurology space.
- Alnylam Pharmaceuticals: Pioneer of RNAi therapeutics and n-Lorem partner. Alnylam partners with n-Lorem to expand technology access to RNAi for nano-rare patients. Comparable because both focus on RNA-targeted precision medicines for rare diseases, and Alnylam's commercial platform represents what an industrialized version of n-Lorem's individualized approach could become.
- Sarepta Therapeutics: Commercial-stage gene therapy company focused on rare neuromuscular diseases. While operating in a different modality (gene therapy vs. ASO), Sarepta is comparable as a leading rare disease therapeutics company that has built infrastructure and regulatory pathways n-Lorem benefits from and aspires to enable for nano-rare populations.
Emerging players
- Stoke Therapeutics: Clinical-stage biotechnology company developing antisense oligonucleotide medicines for severe genetic diseases using TANGO technology for RNA-targeted augmentation. Comparable as an emerging commercial ASO developer focused on rare diseases, representing a more commercially-oriented version of n-Lorem's ASO mission.
- ProQR Therapeutics: Clinical-stage biotechnology company developing RNA-editing therapeutics (Axiomer platform) for rare genetic diseases. Comparable as an emerging RNA-targeted rare disease player; their platform technology could be an alternative or complement to n-Lorem's ASO approach for certain nano-rare mutations.
Others
- Critical Path Institute (C-Path): Public-private partnership that launched the One to Millions initiative with n-Lorem as a founding member. Not a direct peer but a key ecosystem enabler: C-Path develops regulatory tools and standardized pathways that benefit n-Lorem's individualized therapy programs.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat7 records
Key risks5 records
Key highlights7 records
Customer concentration
n-Lorem Foundation social profiles
Digital presencen-Lorem Foundation financial estimates
Financial estimateRevenue estimate
Valuation estimate
n-Lorem Foundation leadership team
Management profileNumber of profiles
Profiles1 record
n-Lorem Foundation funding detail
Funding detailFunding overview
Funding rounds1 record
Investors1 record
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n-Lorem Foundation M&A and investment
M&A and investmentM&A
Investments
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Frequently asked questions about n-Lorem Foundation
What does n-Lorem Foundation do?
n-Lorem Foundation develops and provides personalized experimental antisense oligonucleotide (ASO) medicines, for free for life, to patients with nano-rare genetic diseases (1-30 patients worldwide with the same mutation). Each treatment is designed for an individual patient's unique single-gene mutation affecting the central nervous system, liver, kidney, or eye, leveraging 30+ years of ASO technology expertise developed at Ionis Pharmaceuticals.
Is n-Lorem Foundation a public or private company?
n-Lorem Foundation is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was n-Lorem Foundation founded?
n-Lorem Foundation was founded in 2020. It employs 1 to 10 people.
Where is n-Lorem Foundation based?
n-Lorem Foundation is headquartered in California City, United States, in the North America region.
How does n-Lorem Foundation make money?
Four revenue lines are on record. Charitable Donations and Philanthropy is the primary driver. The others are federal and State Grants, directed Donations and commercial Partnerships - Milestones and Royalties.
Who are n-Lorem Foundation's main competitors?
Direct peers on record are Mila's Miracle Foundation, N=1 Collaborative and EspeRare Foundation. Broad incumbents are Ionis Pharmaceuticals, Biogen, Alnylam Pharmaceuticals and Sarepta Therapeutics. Emerging players are Stoke Therapeutics and ProQR Therapeutics. Critical Path Institute (C-Path) is listed as an others.
Does n-Lorem Foundation have an API?
No public API is recorded for n-Lorem Foundation.
What industry is n-Lorem Foundation in?
n-Lorem Foundation's product category is Personalized Genomic Medicine. Its primary akta.pro industry code is HLAAADAC, Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown), with a secondary code of HLAIAIAN, Rare Pediatric & Congenital Disorder Therapies. Its NAICS code is 541714 and its SIC code is 2834.