Lowy Medical Research Institute

What Lowy Medical Research Institute does

Lowy Medical Research Institute (LMRI) is a nonprofit medical research institute headquartered in La Jolla, California, founded in 2004 and dedicated exclusively to understanding and treating macular telangiectasia type 2 (MacTel), a rare neurodegenerative retinal disease. The institute operates an integrated research platform comprising a global patient registry (the MacTel Project NHOR, with over 2,800 active participants), a multi-phase clinical trials program (including the NT-501 CNTF implant under FDA Priority Review with a March 18, 2025 PDUFA date, the Phase 2a SAFE study, and a Laser Study), a laboratory research program investigating MacTel metabolism, genetics, and stem cell biology, an Eye Donation Program, and a multi-modal imaging service spanning OCT, OCT-angiography, FLIO, microperimetry, and adaptive optics.

LMRI's core technical capabilities include metabolomics research (mass spectrometry and stable isotope tracing of amino acid and sphingolipid metabolism), Müller glia and photoreceptor biology, iPSC-derived retinal cell models, transcriptional profiling of the MacTel zone, non-coding RNA research at the 5q14.3 susceptibility locus, and AI/ML-based retinal image segmentation developed in collaboration with the University of Washington Lee Lab. The institute coordinates more than 30 clinical and research centers across North America, Europe, Australia, and the Middle East, with the Moorfields Eye Hospital Reading Centre providing centralized diagnostic reads and partnerships with the Broad Institute, Scripps Research Institute, and numerous universities supplying specialized scientific expertise.

LMRI's business model is that of a philanthropy-funded nonprofit; it does not charge patients for registry participation or clinical research. Operating revenue is sourced primarily from the Lowy Family and other private philanthropy, supplemented by grants and any future licensing or royalty arrangements tied to therapeutic candidates developed by its commercial partner, Neurotech Pharmaceuticals. Patients and their families are research participants rather than paying customers, while clinical sites serve as research collaborators. Effective leadership is provided by President Martin Friedlander (also a professor at Scripps Research Institute) along with a team of Principal Scientists, a Chief Medical Officer (Simone Tzaridis), a Director of Clinical Trials (Jennifer Trombley), and a Chief of Staff (Lea Scheppke).

Name

Lowy Medical Research Institute

Legal name

Lowy Medical Research Institute

Website

https://lmri.net

Company type

Private

Founded year

2004

Operating status

Operating

Headcount range

11–50 employees

Short description

Lowy Medical Research Institute is a nonprofit medical research institute in La Jolla, California, dedicated to understanding and treating macular telangiectasia type 2 (MacTel) through a global patient registry, multi-phase clinical trials, laboratory research, and advanced retinal imaging.

Ownership category

akta.pro rank

Product category

Ophthalmic Research

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Rare Ophthalmology Disorder Therapies (HLAIAIAK)

akta.pro secondary industries

Real-World Data (RWD) / Real-World Evidence (RWE) & Observational Research Platforms (HLACAOAM), Visual Field & Functional Vision Testing (Perimetry, ERG/VEP, Contrast Sensitivity) (HLAHAJAC)

Keywords

• Ophthalmic research
• Rare disease studies
• Clinical trial services
• Biomedical research
• Retinal disease research

Headquarters

HQ city

La Jolla

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Operations, Technology or R&D, Others

Revenue model

1. Research Funding and Grants: LMRI operates as a nonprofit medical research institute funded primarily through private philanthropy (the Lowy Family), grants, and potentially licensing revenue from research discoveries.
2. Clinical Trial Support: Supporting Neurotech Pharmaceuticals in conducting clinical trials for NT-501 treatment for MacTel.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

LMRI is a nonprofit medical research institute that operates an international clinical research program dedicated to macular telangiectasia type 2 (MacTel), a rare neurodegenerative retinal disease. Core offerings include the MacTel Project Registry (NHOR) tracking over 2800 active participants across 30+ global clinical centers, support for multi-phase clinical trials of candidate therapies (including the NT-501 encapsulated cell therapy implant), laboratory and genetic research into MacTel mechanisms, an eye donation program for tissue-based studies, and advanced retinal imaging services (OCT, OCT-A, FLIO, adaptive optics, microperimetry).

Product overview

The Lowy Medical Research Institute operates a unified research platform focused on macular telangiectasia type 2 (MacTel), a rare neurodegenerative eye disease. The organization runs multiple interconnected programs: the MacTel Project Registry (NHOR) which tracks over 2800 patients globally; a Clinical Trials Program advancing the NT-501 (revakinagene taroretcel) implant through Phase 3; a Laboratory Research Program investigating metabolic and genetic mechanisms; an Eye Donation Program providing critical tissue for research; and Imaging Services utilizing advanced modalities like OCT and FLIO. The primary product candidate is NT-501, an encapsulated cell therapy implant delivering CNTF for sustained retinal neuroprotection, currently under FDA review with a March 2025 PDUFA date.

Differentiator

Problem solved

Functional benefit

Brands

• MacTel Project: International research initiative focused on finding causes and treatments for macular telangiectasia type 2, overseen by LMRI

Products and services

• MacTel Project Registry (NHOR) An international clinical research registry that tracks patients with macular telangiectasia type 2. The registry includes archived retinal pictures, medical information, and vision test results from over 2800 active participants across more than 30 centers globally. It is offered to MacTel patients and serves as the longitudinal data backbone for LMRI's clinical research program.
• NT-501 (revakinagene taroretcel) Implant An encapsulated cell therapy (ECT) implant that delivers ciliary neurotrophic factor (CNTF) for the treatment of chronic retinal diseases including macular telangiectasia type 2. The implant uses Neurotech's ECT platform to provide sustained delivery of CNTF, aiming to slow retinal degeneration. LMRI supports the clinical development of NT-501; the candidate is currently under FDA review with a PDUFA goal date of March 18, 2025.
• Clinical Trials Program LMRI's multi-phase clinical trials program for MacTel treatments, including Phase 1/2 and Phase 3 CNTF (NT-501) trials, a Laser Study, Corneal Nerve Fiber Study, and the SAFE study testing serine supplementation and fenofibrate, with patient enrollment across US, UK, Europe, and Australia clinical sites.
• Laboratory Research Program Basic and translational research focused on understanding the metabolic, genetic, and cellular mechanisms underlying MacTel, including metabolism research, genetics studies (including the 5q14.3 locus), stem cell research, and iPSC-derived retinal cell studies.
• Eye Donation Program An eye donation program for people with MacTel who have died, enabling scientists to study MacTel eyes at the cellular level and correlate tissue findings with participants' archived retinal pictures and medical information.
• Imaging Services Advanced retinal imaging services for MacTel diagnosis, monitoring, and research, including optical coherence tomography (OCT), OCT-angiography, fundus autofluorescence, fluorescence lifetime imaging ophthalmoscopy (FLIO), microperimetry, and adaptive optics imaging, conducted across participating clinical centers and coordinated with the Moorfields Reading Centre.

Quantifiable outcome

• 56.4% rate of reduction in disease progression (Protocol A, p<0.0001) and 29.2% rate of reduction (Protocol B, p=0.021)
• +3 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability6 records

Feature7 records

Partnerships

Six partnerships are on record, tiered core and minor.

• Neurotech PharmaceuticalscoreStrategic or Co-development Partner · 8 November 2024Neurotech Pharmaceuticals and LMRI have collaborated on the development of NT-501 (revakinagene taroretcel) using encapsulated cell therapy to deliver CNTF for MacTel treatment. Neurotech received FDA Priority Review and PDUFA goal date of March 18, 2025 for the BLA.
• Prevent BlindnessminorGTM or Marketing PartnerPrevent Blindness partnered with Neurotech to sponsor a free community webinar about MacTel Type 2 on November 14, 2024 to provide information and education to patients.
• Moorfields Eye Hospital Reading CentrecoreStrategic or Co-development PartnerThe Moorfields Reading Centre evaluates all ophthalmic images collected as part of the MacTel Project Registry and Natural History Observation Study, providing official MacTel diagnosis for participants.
• Broad Institute of MIT and HarvardcoreStrategic or Co-development PartnerBroad Institute scientists including Clary Clish (Senior Director of Metabolomics) and other researchers collaborate on metabolomics and genetics research for MacTel.
• The Scripps Research InstitutecoreStrategic or Co-development PartnerLMRI President Martin Friedlander is Professor at Scripps Research Institute. Many LMRI staff and researchers collaborate with Scripps on retinal research and stem cell technologies.
• Collaborating Universities and Research InstitutionscoreStrategic or Co-development PartnerLMRI works with investigators at universities worldwide including Columbia University, Walter and Eliza Hall Institute, University of Utah, University of Sydney, UC San Diego, University of Washington, University College London, and others for MacTel research.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella advocacy and research organization serving the entire rare disease community. Comparable as a structural peer operating in the rare-disease research/clinical trial ecosystem, although broader in scope than LMRI's single-disease focus.
• Apellis Pharmaceuticals: Commercial retinal disease therapeutics company (Empaveli/Syfovre) operating in macular and retinal degeneration. Comparable as a stakeholder/competitor in the broader retinal disease therapeutics field where LMRI's research outputs eventually land.
• Cystic Fibrosis Foundation: Established rare-disease nonprofit that accelerated translational research and therapeutic development (e.g., Trikafta). Comparable as a benchmark for LMRI's potential evolution into a translational engine that advances therapies from basic science to clinical approval.

Direct peers

• BrightFocus Foundation: Nonprofit funding vision and brain disease research, including grant programs for macular and retinal disease research. Comparable model to LMRI: philanthropy-driven, focused on ophthalmology, supports emerging therapeutics and scientific publications.
• Foundation Fighting Blindness: Nonprofit research foundation funding and driving clinical programs for inherited retinal degenerative diseases. Highly comparable to LMRI as a rare-disease eye-research nonprofit that funds translational science, supports patient registries, and catalyzes clinical trials across a global investigator network.
• Rett Syndrome Research Trust: Focused, family-philanthropy-backed rare disease research foundation driving translational science and clinical trials. Highly comparable operating model to LMRI: small staff, philanthropy-funded, deep disease specialization, active clinical trial portfolio.
• Cure SMA: Family-driven rare disease nonprofit that funded early drug discovery and clinical trials leading to approved SMA therapies. Directly comparable to LMRI in operating model, mission orientation, and scale; representative of how a small rare-disease foundation can translate research into approved treatments.

Others

• Scripps Research Institute: Academic research institute where LMRI's President holds a faculty appointment and where many LMRI staff trained. Comparable as a translational biomedical research counterpart operating in the La Jolla research ecosystem; functions as an enabling/infrastructure partner rather than direct competitor.

Regional players

• Moorfields Eye Hospital / NIHR Biomedical Research Centre: UK-based ophthalmology clinical and research center that operates the Moorfields Reading Centre evaluating all MacTel Project imaging and provides official diagnoses. Direct clinical/infrastructure peer participating in LMRI's MacTel network.

Emerging players

• Neurotech Pharmaceuticals: Clinical-stage biotech developing the NT-501 encapsulated cell therapy implant for MacTel and other retinal diseases. Strategic partner and translational counterpart to LMRI: comparable function in the MacTel program (LMRI supports trials and registry, Neurotech owns commercial rights to the implant).

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/lowy-medical-research-institute-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Lowy Medical Research Institute