Usher III Initiative

What Usher III Initiative does

Usher III Initiative is a 501(c)(3) nonprofit medical research organization founded in 2007 by Cindy Elden, herself a Usher syndrome type III (USH3) patient, and her father Richard Elden. The organization is dedicated to finding treatments, causes, and cures for USH3, the rarest form of Usher syndrome (representing approximately 2% of the ~400,000 Usher patients worldwide), a genetic disorder causing progressive hearing and vision loss leading to deaf-blindness.

Its core asset is BF844, a first-in-class small molecule chaperone therapy that prevents HSP60 from degrading the Clarin-1 protein critical for vision and hearing. BF844 was discovered through research at Case Western Reserve University (Dr. Yoshikazu Imanishi and Dr. Kumar Alagramam) and published in Nature Chemical Biology. The compound is protected by 11+ granted patents spanning the US, Australia, Canada, Europe, Hong Kong, Israel, Japan, and South Africa. Complementary programs include AAV-based gene therapy research at the University of Florida and Columbia University. In October 2024, the organization commenced first-ever human Phase 1 clinical trials for BF844.

The organization operates a donation-and-grant-based revenue model (no products or services are sold), with funding drawn from individual donations via the Kindful platform, foundation grants (notably a $1M TRAP grant from Foundation Fighting Blindness in 2021), and corporate/institutional partnerships. It coordinates a network of academic collaborators, runs a global patient registry engagement program through partnerships with USH Trust and My Retina Tracker, and maintains a Scientific Advisory Board staffed by senior figures from Genentech, Columbia University, Vanderbilt University, and the Retina Foundation of the Southwest. Headcount is 1-10, with offices in Chicago, IL and a contact address in New York.

Name

Usher III Initiative

Legal name

Usher III Initiative

Website

https://usheriii.org

Company type

Private

Founded year

2007

Operating status

Operating

Headcount range

1–10 employees

Short description

Usher III Initiative is a 501(c)(3) nonprofit medical research organization developing BF844, a first-in-class small molecule chaperone therapy for Usher syndrome type III, alongside complementary gene therapy programs, serving the global USH3 patient community of approximately 8,000 individuals.

Ownership category

akta.pro rank

Product category

Rare Disease Medical Research

NAICS

Scientific Research and Development Services (5417)

SIC

Services-Health Services (8000), Patent Owners & Lessors (6794), Services-Social Services (8300)

akta.pro primary industry

Global Health Research, Clinical Trials & Product Development Partnerships (PDPs) (HLAJAOAM)

akta.pro secondary industry

Clinical Development (Phase I–III Trial Design & Execution) (HLAIALAE)

Keywords

• Rare disease research
• Orphan drug development
• Genetic disorder treatment
• Medical research nonprofit
• Patient advocacy foundation

Headquarters

HQ city

Chicago

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales

Revenue model

1. Individual Donations: Tax-deductible donations from individuals supporting Usher III research; donations accepted online via Kindful platform or by mail
2. Foundation Grants: Translational Research Acceleration Program (TRAP) grants; received $1M grant from Foundation Fighting Blindness for pre-IND toxicity studies
3. Corporate/Institutional Partnerships: Partnerships with pharmaceutical companies for drug development and clinical trials

Go-to-market motion2 records

Distribution channels3 records

Marketing channels6 records

Core offering

Usher III Initiative is a nonprofit research organization developing BF844, a first-in-class small molecule chaperone therapy designed to treat Usher syndrome type III caused by the N48K mutation in the CLRN1 gene. The compound works by preventing HSP60 from degrading Clarin-1 protein, which is essential for vision and hearing. The organization also coordinates patient registry enrollment and provides educational resources to the USH3 community.

Product overview

Usher III Initiative is a nonprofit medical research organization developing BF844, a first-in-class small molecule therapy for Usher syndrome type III. The organization functions as a research consortium that funds and coordinates academic and industry research efforts, supports patient registries for clinical trial readiness, and provides educational resources to the Usher community. The core product is the BF844 drug candidate, with complementary patient support services including registry enrollment assistance and research information resources.

Differentiator

Problem solved

Functional benefit

Brands

• BF844: Small molecule compound (BF844) being developed as a treatment for Usher syndrome type III caused by the N48K mutation in the CLRN1 gene

Products and services

• BF844 First-in-class small molecule chaperone therapy being developed for Usher syndrome type III caused by the N48K mutation in the CLRN1 gene. The compound prevents HSP60 from breaking down Clarin-1 protein essential for vision and hearing. First human clinical trials commenced in October 2024.
• Patient Registry Enrollment Support Coordination and support for patient participation in global registries including My Retina Tracker (Foundation Fighting Blindness) and USH Trust (Usher Syndrome Coalition) to facilitate clinical trial recruitment and research data collection for Usher III patients.
• Patient Education and Research Resources Educational materials and information about Usher syndrome, genetic testing resources, and research developments provided to patients, families, and the broader community to support diagnosis, awareness, and engagement with USH3 research progress.

Quantifiable outcome

• BF844 showed no overt toxicity in pre-clinical safety tests including genotoxicity, cardiac safety, and cellular assays
• +2 more outcomes

Named customers4 records

Segments3 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Nine partnerships are on record, tiered core and minor.

• Foundation Fighting BlindnesscoreStrategic or Co-development Partner · 16 August 2024Partnership to launch a retrospective natural history study for Usher Syndrome Type 3. This study will map average disease progression, identify ideal point for therapeutic intervention, quantify confirmed USH3 patients, and define Phase-II clinical trial outcomes.
• Case Western Reserve UniversitycoreStrategic or Co-development PartnerKey research institution led by Dr. Kumar Alagramam and Dr. Yoshikazu Imanishi conducting BF844 compound testing, transgenic mouse development, and protein interaction studies.
• University of FloridacoreStrategic or Co-development PartnerBill Hauswirth's lab conducting gene therapy research for USH3 in the eye; developing AAV vectors for Clarin1 gene delivery.
• Columbia UniversitycoreStrategic or Co-development PartnerDr. Lawrence Lustig's lab (Chair, Department of Otolaryngology/Head and Neck Surgery) conducting gene therapy for hearing preservation in USH3 mice.
• Biotic ArtlabminorStrategic or Co-development PartnerMedical communications studio developing creative visual solutions (illustration, animation, infographics) for Usher III Initiative's science and patient education materials.
• Usher Syndrome CoalitioncoreStrategic or Co-development PartnerPartnership for USH Trust patient registry, the world's largest Usher syndrome registry, providing secure platform for patients to connect, stay informed, and participate in clinical trials.
• National Institute on Deafness and Other Communication Disorders (NIDCD)minorStrategic or Co-development PartnerResource partner providing information about Usher syndrome, its progression, and genetic causes for patient education.
• Dana-Farber Cancer InstituteminorStrategic or Co-development PartnerDr. Julia Hayes serves on Board of Directors; Medical Oncologist providing oncology expertise.
• Retina Foundation of the SouthwestminorStrategic or Co-development PartnerDr. David Birch serves on Scientific Advisory Board; Scientific Director leading the Rose-Silverthorne Retinal Degenerations Laboratory.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella rare disease advocacy organization. Provides policy, research grants, and patient registry infrastructure. Comparable nonprofit rare disease operating model though at much broader scale across 7,000+ diseases.
• Friedreich's Ataxia Research Alliance (FARA): Patient-driven nonprofit funding research for a single rare genetic disease (Friedreich's ataxia). Closely analogous to Usher III Initiative's single-disease focus and patient registry-driven clinical trial recruitment model.
• Spark Therapeutics: Pioneer in gene therapy for inherited retinal diseases (Luxturna for RPE65 mutations). Acquired by Roche. Comparable focus area with established regulatory and commercial pathway in USH-adjacent orphan ophthalmology indications.
• ProQR Therapeutics: Clinical-stage biotech developing RNA therapies for inherited retinal diseases including Usher syndrome. Highly comparable therapeutic focus on USH2A (Ultevursen) targeting the same disease family with a different modality.
• Cure SMA: Established rare disease nonprofit that successfully drove an entire therapeutic pipeline (Spinraza, Zolgensma, Evrysdi) from research funding to approved drugs. Highly comparable nonprofit-driven rare disease drug development model with broader resources and established track record.

Direct peers

• Foundation Fighting Blindness: Largest private funder of retinal degenerative disease research ($816M+ raised). Directly partners with Usher III Initiative via $1M TRAP grant and natural history study. Highly comparable nonprofit research-funding model targeting inherited retinal diseases.
• Retina Foundation of the Southwest: SAB partner of Usher III Initiative. Scientific Director Dr. David Birch leads the Rose-Silverthorne Retinal Degenerations Laboratory. Conducts clinical research on inherited retinal diseases including Usher syndrome.
• Usher Syndrome Coalition: Operates the USH Trust patient registry that Usher III Initiative relies on. Closely comparable mission (Usher syndrome support and research), though broader in scope across all Usher types (1, 2, and 3).

Emerging players

• Editas Medicine: Clinical-stage gene editing company developing therapies for inherited retinal diseases. Comparable in targeting the same ophthalmology rare disease space via gene therapy/editing approaches that compete with or complement BF844.

Others

• Blueprint Genetics: SAB member Dr. Eeva-Marja Sankila serves as Clinical Consultant. Provides genetic testing services for inherited eye diseases, including Usher syndrome, directly supporting the diagnostic pathway Usher III Initiative relies on for patient identification.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xtwitter.com/UsherIII
• Facebookfacebook.com/Usher3Initiative
• Instagraminstagram.com/usheriii
• LinkedInlinkedin.com/company/usher-iii-initiative-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles18 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Usher III Initiative