Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

What Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) does

LDRTC (Lysosomal and Rare Disorders Research and Treatment Center, Inc.) is a 501(c)(3) nonprofit clinical research organization founded in 2013 by Dr. Ozlem Goker-Alpan and headquartered at 3702 Pender Drive, Suite 170, Fairfax, Virginia, where it operates a single-site facility combining patient care, translational research, and sponsored clinical trials under one roof. It serves patients diagnosed with lysosomal storage disorders — primarily Gaucher, Fabry, Pompe, MPS, and Niemann-Pick disease — through enzyme replacement therapy infusions, genetic evaluation, screening programs, and enrollment in registries and treatment trials. The center maintains translational capabilities anchored by an on-site mass spectrometry laboratory, pharmacological chaperone screening using patient-derived primary cell lines, and a first-of-kind 3D bioprinted bone model for Gaucher disease developed with Penn State.

LDRTC's core services span three integrated lines: Clinical Care and Treatment Services (infusions, injections, genetic evaluation, echocardiogram/EKG/PFT diagnostics), a Translational Research Unit (biomarker and 'omics discovery, personalized drug screening), and Clinical Trials Research (registries, sponsor-initiated treatment and non-treatment studies, investigator-initiated studies). The clinical trials program contributed patient data to FDA approvals of Elfabrio (Fabry, May 2023) and Pombiliti/Opfolda (Pompe, October 2023), and in 2023 opened seven new studies, including two gene therapy studies. Supporting the core model are the annual GRIDS Symposium (9th edition in 2024 with 200+ international attendees), quarterly CME webinars with CheckRare and AffinityCE, an HCP fellowship, and patient education meetings across the major LSD advocacy communities.

Revenue mechanics combine three streams: clinical service fees from patient care, grants and charitable contributions (its 501(c)(3) support base), and per-patient/per-study revenue from pharmaceutical-sponsored clinical trials. Pricing for clinical services is not publicly disclosed and varies by insurance arrangement; LDRTC also serves on the scientific advisory boards of multiple pharma companies and partners with academic institutions (Howard University, Penn State). Operating geographies are concentrated in the Northern Virginia / D.C. Metro area, with telemedicine providing limited geographic reach and the GRIDS symposium providing international brand visibility without international clinical delivery.

Name

Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

Legal name

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Website

https://lysosomalcenter.org

Company type

Private

Founded year

2013

Operating status

Operating

Headcount range

11–50 employees

Short description

LDRTC is a 501(c)(3) nonprofit clinical research center in Fairfax, Virginia that integrates specialized care, translational biomarker research, and pharmaceutical-sponsored clinical trials for patients with lysosomal storage disorders including Gaucher, Fabry, Pompe, MPS, and Niemann-Pick disease.

Ownership category

akta.pro rank

Product category

Rare Disease Treatment Services

NAICS

Scientific Research and Development Services (5417)

SIC

Services-Offices & Clinics Of Doctors Of Medicine (8011)

akta.pro primary industry

Site Data Management Support (EDC/Source/SDV Readiness) (HLAGACAL)

akta.pro secondary industry

Laboratory Developed Tests (LDTs) & Specialized Clinical Lab Assays (HLAAALAN)

Keywords

• Rare disease treatment
• Lysosomal storage disorders
• Clinical research services
• Translational research
• Genetic disease care

Headquarters

HQ city

Fairfax

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

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

Revenue model

1. Clinical Services: Revenue from patient care services including infusions, injections, genetic evaluation, and clinical procedures at their Northern Virginia facility.
2. Grants and Charitable Funding: As a nonprofit 501(c)(3) organization, LDRTC relies on grants to maintain programs, services, and fulfill its mission. Form 990s are filed with the IRS and made publicly available.
3. Clinical Trial Participation: Revenue from conducting sponsored clinical trials for pharmaceutical companies and research studies.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels6 records

Core offering

LDRTC is a nonprofit 501(c)(3) organization founded in 2013 by Dr. Ozlem Goker-Alpan that delivers specialized clinical care for patients with lysosomal storage disorders (including Gaucher, Fabry, Pompe, MPS, and Niemann-Pick diseases) and related rare conditions. The center integrates patient care, translational research, and clinical trial operations in a single facility located in Fairfax, VA, with a staff of approximately 22 employees.

Product overview

LDRTC operates as a unified clinical research center offering integrated care for lysosomal storage disorders and rare genetic diseases. The core offering combines Clinical Care and Treatment Services with a Translational Research Unit and Clinical Trials Research program, all under one roof in Fairfax, Virginia. Supporting the core services are educational programs including the annual GRIDS Symposium, CME Webinars, HCP Fellowship training, and internship programs. The center also provides specialized infusion services, genetic evaluation, and maintains an on-site Mass Spectrometry Laboratory. This integrated model enables bench-to-bedside research translation directly benefiting patient care.

Differentiator

Problem solved

Functional benefit

Products and services

• Specialty Clinical Care for Lysosomal Storage Disorders
• Translational Research Program
• Clinical Trials for Rare Diseases

Quantifiable outcome

• Over 20 patients' data contributed to FDA approvals of Elfabrio for Fabry disease (May 2023) and Pombiliti/Opfolda for Pompe disease (October 2023)
• +2 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles1 record

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Four partnerships are on record, tiered core.

• Howard University Hospital and College of MedicinecoreStrategic or Co-development PartnerLDRTC collaborated with Howard University Hospital and College of Medicine in Washington DC on a large-scale screening study to investigate the incidence of three lysosomal storage disorders (Gaucher, Pompe, and Fabry diseases) in a cohort of mostly urban-dwelling African-Americans. The study identified novel mutations and highlighted higher incidence of abnormal enzyme levels in the target population.
• Penn State University (Dr. Ozbolat's Lab)coreStrategic or Co-development PartnerCollaborated with Dr. Ozbolat from Penn State University to develop the first 3D human model of Gaucher disease using aspiration-assisted freeform bioprinting technology. This collaboration created a platform for decoding cellular basis of developmental bone abnormalities and personalized drug screening.
• CheckRare and AffinityCEcoreStrategic or Co-development PartnerLDRTC co-hosted quarterly CME/CE webinar series on lysosomal storage diseases with CheckRare (a leading publisher and learning platform focused on rare diseases) and AffinityCE (an accredited medical education company). The collaboration provides continuing education for healthcare professionals on LSDs.
• Pharmaceutical CompaniescoreStrategic or Co-development PartnerLDRTC serves on scientific advisory boards of multiple pharmaceutical companies and conducts clinical trials for pharmaceutical sponsors. The organization has participated in studies contributing to FDA approvals of treatments for Fabry and Pompe diseases.

Scale indicators6 records

Recent moves7 records

Expansion highlights6 records

Emerging players

• Ultragenyx Pharmaceutical: Ultragenyx develops therapies for rare and genetic diseases including several LSDs (e.g., Crysvita for XLH, Dojolvi for LC-FAOD). It is a smaller, more focused LSD/rare disease biotech than the top pharma incumbents, with gene therapy programs that align with LDRTC's translational research and clinical trial interests.
• Chiesi Global Rare Diseases: Chiesi developed Elfabrio (pegunigalsidase alfa) for Fabry disease, which received FDA approval in May 2023 based on studies that enrolled LDRTC patients starting in 2013. As the sponsor of one of LDRTC's most consequential trial contributions, Chiesi is a direct partner peer in the lysosomal Fabry disease niche.
• REGENXBIO: REGENXBIO is developing gene therapies for rare diseases including MPS I, MPS II, and other LSDs using its NAV platform. As gene therapy trials grow as a share of LDRTC's portfolio (2 new gene therapy studies opened in 2023), REGENXBIO represents a likely sponsor peer with overlapping scientific and operational interests in advanced LSD therapeutics.

Others

• National Organization for Rare Disorders (NORD): NORD is the leading U.S. patient advocacy nonprofit for rare diseases, operating research programs, patient registries, and educational initiatives across the same disease communities LDRTC serves. It is a thematically related ecosystem participant rather than a direct competitor, but LDRTC collaborates with similar advocacy organizations in LSD patient outreach.
• National Gaucher Foundation: The National Gaucher Foundation is a patient advocacy and support organization specifically for the Gaucher disease community that LDRTC actively serves. It is an ecosystem participant that supports patient education and clinical trial recruitment in LDRTC's primary Gaucher patient segment.
• Rare Disease Clinical Research Network (RDCRN): RDCRN is an NIH/NCATS-funded network of research consortia across rare diseases including LSDs, providing a federated clinical trial infrastructure and patient registry. It is a related research ecosystem participant that competes with and complements LDRTC's translational and clinical research activities in overlapping LSD populations.

Broad incumbents

• Sanofi Genzyme: Sanofi's rare disease franchise (formerly Genzyme) is the largest commercial player in lysosomal storage disorders, with approved enzyme replacement therapies for Fabry (Fabrazyme), Gaucher (Cerezyme), and Pompe (Myozyme/Lumizyme) diseases. LDRTC conducts sponsored trials for these and competing therapies, and serves on pharma scientific advisory boards alongside Sanofi-affiliated investigators, making it the most relevant commercial incumbent in the LSD treatment space.
• BioMarin Pharmaceutical: BioMarin is a rare disease-focused biopharma with multiple enzyme replacement products (Aldurazyme for MPS I, Naglazyme for MPS VI, Vimizim for MPS IVA). BioMarin competes with and complements LDRTC's work in MPS and adjacent LSD populations, and represents a likely sponsor partner for any future MPS-related clinical trials.
• Takeda Pharmaceutical Company: Takeda, through its 2019 Shire acquisition, became a major lysosomal disease franchise player with Replagal (Fabry) and Vpriv (Gaucher). Takeda is both a potential LDRTC trial sponsor and a competitor for patient referrals in the same disease areas, with significantly larger commercial and clinical operations across multiple LSDs.

Direct peers

• Amicus Therapeutics: Amicus is a pure-play lysosomal disease biotech whose Galafold (Fabry) and Pombiliti+Opfolda (Pompe) combination therapy received FDA approval in October 2023 with direct LDRTC patient contribution. As a focused LSD developer that LDRTC works with on pivotal trials, Amicus is among the closest direct peers in the lysosomal disease niche.

Market position

Strengths3 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/ldrtc

Revenue estimate

Valuation estimate

Number of profiles

Profiles1 record

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 Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)