Orphan Disease Center | University of Pennsylvania

Name

Orphan Disease Center | University of Pennsylvania

Legal name

Orphan Disease Center

Website

https://orphandiseasecenter.med.upenn.edu

Company type

Private

Founded year

2011

Operating status

Operating

Headcount range

1–10 employees

Ownership category

akta.pro rank

Product category

Medical Research Funding

NAICS

Voluntary Health Organizations (813212), Grantmaking and Giving Services (81321)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Health & Medical Research Grantmaking Foundations (BPAGAKAL)

akta.pro secondary industries

Rare Disease & Special Needs Support Organizations (BPAGACAM), Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

Keywords

• Rare disease research
• Grant funding programs
• Orphan disease therapy
• Translational research
• Patient advocacy partnerships

Headquarters

HQ city

Philadelphia

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, Infrastructure, Marketing or Sales

Revenue model

1. Grant Funding: The Orphan Disease Center provides grant funding to researchers for rare disease research. Since 2011, the center has funded $37.6 million in rare disease research through various grant programs including the Million Dollar Bike Ride grants, JumpStart program grants, and Program of Excellence grants.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels5 records

Core offering

The Orphan Disease Center (ODC) at the University of Pennsylvania School of Medicine, co-affiliated with Children's Hospital of Philadelphia (CHOP), facilitates and funds rare disease research and develops transformative therapies for orphan diseases. It distributes over 50 grant opportunities annually across 30+ disease areas and has disbursed $37.6 million in rare disease research funding since 2011. Core offerings include a grant opportunities program, the JumpStart program for emerging rare diseases, Programs of Excellence (e.g., CDKL5, MPS), and the Million Dollar Bike Ride fundraising event.

Product overview

The Orphan Disease Center at the University of Pennsylvania is a research funding organization that operates as a platform of interconnected programs to facilitate and fund rare disease research. The core offerings include the Grant Opportunities Program (offering 50+ annual grants across 30+ disease areas), the JumpStart Program for emerging rare diseases, and the Million Dollar Bike Ride fundraising initiative. Specialized Programs of Excellence (PoE) focus on specific diseases including CDKL5 Deficiency Disorder and Mucopolysaccharidoses. The center connects researchers globally with patient advocacy groups and provides funding, educational resources, and therapeutic development support for over 7,000 distinct orphan diseases affecting over 25 million Americans.

Differentiator

Problem solved

Functional benefit

Products and services

• Grant Opportunities Program Annual grant funding program providing over 50 grant opportunities across 30+ rare disease areas to academic researchers globally and within the Penn/CHOP community. Since 2011, the program has funded $37.6 million in rare disease research.
• JumpStart Program

Quantifiable outcome

• Over $37.6 million in rare disease research funded since 2011
• +3 more outcomes

Named customers6 records

Segments3 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Six partnerships are on record, tiered core, major and minor.

• Loulou FoundationcoreStrategic or Co-development Partner · 1 January 2016The ODC has partnered with the Loulou Foundation to create the CDKL5 Program of Excellence (PoE) within the ODC. The PoE is tasked with driving the development of effective therapeutic strategies for the treatment of CDKL5 Deficiency Disorder (CDD). Since 2016, the ODC and the Loulou Foundation have awarded and managed more than 75 grants totaling over $11 million to institutions around the world. This research support has advanced understanding of CDKL5 under normal and pathological conditions, generated necessary tools for disease modeling and drug discovery, and advanced therapeutic development.
• Children's Hospital of Philadelphia (CHOP)coreStrategic or Co-development PartnerThe Orphan Disease Center is part of both the University of Pennsylvania's School of Medicine and Children's Hospital of Philadelphia. This dual affiliation provides access to world-class research infrastructure and enables collaboration between two leading pediatric healthcare institutions.
• University of Pennsylvania School of MedicinecoreStrategic or Co-development PartnerThe Orphan Disease Center is part of the University of Pennsylvania's School of Medicine, providing institutional affiliation, research infrastructure, and access to Penn's Gene Therapy Program capabilities.
• CDKL5 AlliancemajorStrategic or Co-development PartnerPatient advocacy group listed as a CDKL5 patient advocacy organization partnering with ODC's CDKL5 research programs.
• International Foundation for CDKL5 Research (IFCR)majorStrategic or Co-development PartnerPatient advocacy organization listed as a partner in CDKL5 research ecosystem.
• Telethon Kids InstituteminorStrategic or Co-development PartnerAustralian research institution listed as a CDKL5 patient advocacy group partner.

Scale indicators8 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• EveryLife Foundation for Rare Diseases: EveryLife is a nonprofit that funds rare disease research and policy advocacy, with a comparable focus on accelerating therapeutic development for underserved orphan diseases. Both organizations serve as funders and conveners for the rare disease research ecosystem.
• National Organization for Rare Disorders (NORD): NORD is the leading US rare disease patient advocacy and research-funding nonprofit, operating grant programs and patient registries analogous to ODC's Programs of Excellence. It is the closest direct peer in mission and funding-model terms.
• ALS Association: The ALS Association is the leading disease-specific foundation funding ALS research — directly comparable to ODC's newly established ALS Research Program. Both organizations fund gene therapy and translational research for ALS.
• Parent Project Muscular Dystrophy (PPMD): PPMD funds Duchenne muscular dystrophy research through grants and patient-driven partnerships, mirroring ODC's model of co-funding disease-specific Programs of Excellence with patient advocacy foundations.
• CureSanfilippo Foundation: A disease-specific patient advocacy foundation that funds MPS III (Sanfilippo syndrome) research through grants — directly comparable to ODC's MPS Program of Excellence and the patient-foundation co-funding model.
• Cure SMA: Cure SMA is a disease-specific patient advocacy foundation that funds SMA research grants and partners with academic researchers — directly comparable to ODC's disease-specific Programs of Excellence model (e.g., CDKL5 PoE).

Broad incumbents

• Muscular Dystrophy Association (MDA): MDA is a much larger, established neuromuscular-disease nonprofit that funds research, operates care centers, and runs advocacy campaigns. It overlaps with ODC's ALS program but as a broader portfolio incumbent rather than a specialist.
• Sanford Burnham Prebys Medical Discovery Institute: A broader academic research institute with established rare disease research programs (e.g., in LSDs). Comparable as an academic-affiliated entity conducting and funding translational rare disease research, though at much larger scale.

Others

• NIH Office of Rare Diseases Research (ORDR / NCATS): The federal funder of rare disease research, comparable to ODC in mandate but vastly larger in scale. Acts as an ecosystem participant and indirect funder for many of the researchers ODC supports.

Regional players

• CHOP Foundation / Children's Hospital of Philadelphia Research Institute: CHOP is ODC's parent affiliate and operates a parallel pediatric research institute with grant programs. It is comparable as a pediatric rare disease research funding body within the same geographic and institutional ecosystem.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/orphan-disease-center-university-of-pennsylvania

Revenue estimate

Valuation estimate

Number of profiles

Profiles1 record

Funding overview

Funding rounds

Investors

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M&A

Investments

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