University of Florida Center for NeuroGenetics

What University of Florida Center for NeuroGenetics does

The University of Florida Center for NeuroGenetics (CNG) is an academic research center within the University of Florida College of Medicine, established in 2017 and headquartered at the Cancer Genetics Research Complex in Gainesville, Florida. The Center integrates molecular, genetic, and clinical approaches to investigate the causes of neurodegenerative disease and to develop effective treatment strategies, with particular focus on repeat expansion disorders including amyotrophic lateral sclerosis (ALS), myotonic dystrophy types 1 and 2, spinocerebellar ataxias, Huntington's disease, and Alzheimer's disease. It is organized around five PI-led faculty labs — the Ranum, Nguyen, Fujii, Swanson, and Wang Labs — and operates a specialized sub-unit, the Wellstone Center, dedicated to myotonic dystrophy research with three structured projects and supporting cores.

The Center's scientific portfolio rests on several proprietary discoveries. These include the foundational discovery of Repeat Associated Non-AUG (RAN) translation by the Ranum Lab, the development of a unique C9orf72 BAC transgenic mouse model that closely mimics human ALS/FTD symptoms, a novel therapeutic application of the FDA-approved Type 2 diabetes drug metformin to reduce RAN protein levels in C9orf72-linked ALS, human-derived antibody immunotherapy targeting mutant RAN proteins, CRISPR/Cas9-nickase approaches to shrink expanded repeats in myotonic dystrophy, and research on ribosomal translation fidelity led by the Fujii Lab. Research is supported by a Brain Donation Program and Blood and Tissue Bank that provide access to patient-derived biological samples.

The Center does not sell commercial products or services. Its business model is non-commercial academic research: activities are funded through federal grants (NIH including NINDS, Department of Defense), philanthropic awards (Chan Zuckerberg Initiative's $2.5 million Ben Barres Early Career Acceleration Award in 2018 to Eric Wang), and disease-focused foundations (Target ALS, ALS Association, Packard Center, Myotonic Dystrophy Foundation, National Ataxia Foundation, Muscular Dystrophy Association), supplemented by donations routed through the University of Florida. Its 'customers' are patients participating in clinical research, graduate students and postdoctoral fellows receiving training, and the broader scientific community consuming its publications and mouse model reagents. The Center is part of the public University of Florida state university system and has no independent equity ownership, venture capital, or private equity involvement.

Name

University of Florida Center for NeuroGenetics

Legal name

University of Florida Center for NeuroGenetics

Website

https://neurogenetics.med.ufl.edu

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

The University of Florida Center for NeuroGenetics is an academic research center within the UF College of Medicine that integrates molecular, genetic, and clinical approaches to study and develop treatments for repeat expansion neurodegenerative diseases including ALS, myotonic dystrophy, Huntington's, Alzheimer's, and spinocerebellar ataxias.

Ownership category

akta.pro rank

Product category

Academic Neurogenetics Research

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Research and Development in the Physical, Engineering, and Life Sciences (54171)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Medical Laboratories (8071)

akta.pro industry

PRIMARYHLAKAIAO

Neurogenetics & Rare Neurologic Diseases

Healthcare & Life Sciences/Specialist Medical Practice/Neurology

SECONDARYHLAAACAH

Neurology/CNS Gene Therapies

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAKAIAD

Neuromuscular Medicine (ALS, Myasthenia, Neuropathy)

Healthcare & Life Sciences/Specialist Medical Practice/Neurology

SECONDARYHLAIACAC

Neurology & CNS Specialty Pharmaceuticals

Healthcare & Life Sciences/Pharmaceuticals/Specialty Pharmaceuticals

SECONDARYEDAHAAAC

Public Research Universities with Academic Medical Centers (AMCs)

Education/Higher Education (Colleges & Universities)/Public Research Universities

Keywords

• Neurogenetics research
• Neurodegenerative disease research
• Molecular neuroscience research
• Translation research
• Clinical research

Headquarters

HQ city

Gainesville

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations

Distribution channels4 records

Marketing channels7 records

Core offering

The University of Florida Center for NeuroGenetics is an academic research center within the UF College of Medicine that investigates the genetic and molecular mechanisms underlying neurodegenerative diseases, including repeat-associated non-AUG (RAN) translation in ALS, FXTAS, polyglutamine diseases, and tauopathies. The center develops disease models, conducts clinical and translational research, and operates a brain donation program to support investigator-initiated and collaborative studies.

Product overview

The University of Florida Center for NeuroGenetics (CNG) is a research center within the College of Medicine that integrates molecular, genetic, and clinical approaches to define the causes of neurodegenerative disease and develop effective treatment strategies. The CNG does not offer commercial products but conducts research across multiple focus areas including Amyotrophic Lateral Sclerosis (ALS), Myotonic Dystrophy (DM) Types 1 & 2, Spinocerebellar Ataxias (SCAs), Huntington's Disease, and Alzheimer's Disease. The center provides research collaboration opportunities, clinical trial participation, brain donation programs, and blood/tissue banking services. Research services include laboratory research in repeat expansion diseases, gene discovery, and therapeutic development.

Differentiator

Problem solved

Functional benefit

Brands

• Wellstone Center: A specialized center within CNG focused on myotonic dystrophy research with three main projects covering DM2 pathomechanisms, DM1 therapeutic efficacy, and biological determinants of disease variability.

Products and services

• Neurogenetics Research Programs Investigator-initiated basic and translational research programs focused on the genetic and molecular mechanisms of neurodegenerative diseases, including repeat-associated non-AUG (RAN) translation in ALS, FXTAS, polyglutamine diseases, and tauopathies. Targets academic researchers and clinicians.
• Clinical Trials in Neurodegenerative Disease Clinical research and trial infrastructure for patients with neurodegenerative diseases, embedded within the University of Florida College of Medicine. Targets affected patients and clinical collaborators.
• Brain Donation Program Tissue donation program that collects and provides post-mortem brain tissue for neurodegenerative disease research, supporting investigators studying disease mechanisms. Targets patients and families willing to donate, and the research community.
• Disease Model Development (CRISPR/Cas9 Mouse and Cellular Models) Development of CRISPR/Cas9-based mouse and patient-derived cellular models of neurodegenerative diseases for use in academic research collaborations. Targets neuroscience and genetics research laboratories.

Quantifiable outcome

• Metformin reduced RAN protein levels in mouse models of C9orf72 ALS, improved behavior, reduced brain inflammation, and increased motor neuron survival
• +2 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Nine partnerships are on record, tiered core and minor.

• Myotonic Dystrophy FoundationcoreStrategic or Co-development Partner · 11 May 2023Provides fellowship funding to CNG researchers including Emma Shea, supporting research on myotonic dystrophy therapeutic development.
• McGill UniversityminorStrategic or Co-development Partner · 4 September 2020Collaboration with McGill University biochemist Nahum Sonenberg, PhD, on metformin research. Sonenberg's discovery of metformin's benefits in Fragile X syndrome inspired the ALS study.
• Entrada TherapeuticscoreStrategic or Co-development Partner · 4 September 2020Collaboration with Entrada Therapeutics on DM1 therapeutic research project using CRISPR/Cas9-nickase approaches to shrink expanded repeats.
• NeurimmunecoreStrategic or Co-development Partner · 9 December 2019Collaboration on developing human-derived antibodies targeting RAN proteins in C9orf72-linked ALS and frontotemporal dementia. Neurimmune's discovery of therapeutic antibodies combined with UF's research expertise to advance immunotherapy approach.
• BiogencoreStrategic or Co-development Partner · 9 December 2019Collaborated on antibody therapy research for genetic form of ALS and FTD, leveraging Biogen's pharmaceutical development capabilities alongside UF's preclinical research.
• Johns Hopkins UniversitycoreStrategic or Co-development Partner · 9 December 2019Collaborating institution in Neuron publication on antibody therapy for ALS/FTD, contributing to the multicenter research effort.
• Evelyn F. and William L. McKnight Brain InstitutecoreStrategic or Co-development PartnerCo-sponsor of Fifth International Brainstorm Symposium and research partner providing institutional infrastructure and support.
• Norman Fixel Institute for Neurological DiseasescoreStrategic or Co-development PartnerCo-sponsor of Fifth International Brainstorm Symposium and collaborative partner in neurological disease research at UF.
• Center for Translational Research in Neurodegenerative Disease (CTRND)coreStrategic or Co-development PartnerCo-sponsor of Fifth International Brainstorm Symposium and partner in translational research efforts.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Denali Therapeutics: Biotech developing therapeutics for neurodegeneration with programs in ALS, Alzheimer's, and other CNS indications. Comparable disease focus with stronger BBB delivery platform and clinical-stage assets.
• Biogen: Major biopharma developing therapies for ALS, FTD, and other neurodegenerative diseases, and a direct strategic partner of CNG on the C9orf72 antibody program. Overlapping disease focus makes it the most comparable large incumbent, though Biogen's portfolio extends well beyond repeat expansion disorders.
• Ionis Pharmaceuticals: Established antisense oligonucleotide leader with programs in ALS, Huntington's, and other neurological indications. Comparable in targeting repeat expansion disease biology at the RNA level, though with a much broader and more mature pipeline.
• Alnylam Pharmaceuticals: RNAi therapeutics pioneer expanding into CNS indications including neurodegenerative diseases. Comparable modality (RNA-targeted intervention) with greater commercial scale and CNS delivery investment.

Direct peers

• Wave Life Sciences: Clinical-stage RNA editing company with multiple programs in Huntington's disease, DM1, and ALS targeting repeat expansion mutations. Closely comparable in therapeutic category (nucleic acid-based interventions for repeat expansion disorders).
• ProQR Therapeutics: RNA editing company developing Axiomer technology for genetic neurological diseases including repeat expansion disorders. Closely comparable therapeutic modality and disease focus, though earlier-stage.
• Entrada Therapeutics: Direct collaboration partner of CNG on CRISPR/Cas9-nickase repeat-shrinking approaches for DM1. Targets the same repeat expansion biology and intracellular delivery challenges, making it the most directly comparable peer in therapeutic modality.

Others

• The Jackson Laboratory: Independent genetics and genomics research institution that distributes mouse models and provides research services globally. Comparable in supporting the neurodegenerative genetics research ecosystem, including mouse model distribution that parallels CNG's BAC model offering.

Emerging players

• Avidity Biosciences: Antibody-RNA conjugate developer with DM1 and other neuromuscular disease programs. Comparable in targeting myotonic dystrophy and using antibody-mediated delivery, though with a more commercial-stage pipeline.

Regional players

• Massachusetts General Hospital Neurogenetics Program: Academic neurogenetics program at a major AMC with overlapping research in repeat expansion diseases and neurodegenerative genetics. Closest academic peer in terms of mission, structure, and disease focus.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/UFMedicine
• Facebookfacebook.com/ufdrgator
• LinkedInlinkedin.com/company/uf-center-for-neurogenetics

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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