Spinal Muscular Atrophy Foundation - Grant Information & Application Details

Quick Stats

Annual Giving: $2.5 million (2023 charitable disbursements)
Success Rate: Not publicly disclosed (invitation-only process)
Decision Time: Variable (letter of intent reviewed before invitation to apply)
Grant Range: $1.65 million - $15 million (based on recent awards)
Geographic Focus: International (focus on SMA research institutions)

Contact Details

Website: https://smafoundation.org Phone: 646-253-7100 Email: info@smafoundation.org Research Funding Inquiries: research@smafoundation.org Mailing Address: SMA Foundation 970 W Broadway STE E PMB 140 Jackson, WY 83001

New York Office: 888 Seventh Ave Ste 400 New York, NY 10019

Overview

The Spinal Muscular Atrophy (SMA) Foundation was established in 2003 by Loren Eng and Dinakar Singh, parents of a child with SMA. Operating as a unique blend of non-profit, venture capital, and biotech entities, the Foundation has become the world's leading funder of SMA research. Since its inception, the Foundation has invested approximately $150 million in basic, translational, and clinical research, plus an additional $30 million in developing critical research tools and drug discovery assets. With total assets of $33.7 million as of 2023, the Foundation focuses on late translational research and clinical trials that will have immediate impact on treatment development. The Foundation has been recognized with the Child Neurology Foundation's Advocacy Award of Merit and the Giblin Foundation Award, and has been instrumental in the development of three FDA-approved SMA treatments.

Funding Priorities

Grant Programs

Research Funding Program (Invitation Only)

Focus on late translational research or clinical trials with immediate impact potential
Principal investigators only
Requires letter of intent submission to research@smafoundation.org
Grant applications accepted by invitation only following letter of intent review
Recent awards range from $1.65 million to $15 million
Application method: Rolling basis via letter of intent, followed by invitation to submit full proposal

Notable Recent Awards:

Columbia University Center for Motor Neuron Biology and Disease: Up to $15 million
PTC Therapeutics: $8.5 million for small molecule development collaboration
Regeneron Pharmaceuticals: $1.65 million for new SMA animal models

Priority Areas

The Foundation concentrates its funding on work that will have immediate impact on:

Late Translational Research: Advanced preclinical studies ready to move toward clinical applications
Clinical Trials: Studies directly testing SMA treatments in human subjects
Drug Discovery: Small molecule development and therapeutic screening
Research Tools & Models: Development of validated research tools, in vitro and in vivo disease models for use by the broader research community
Regenerative Medicine: Novel approaches to restore or replace damaged motor neurons
Academic-Industry Collaborations: Partnerships between universities and pharmaceutical/biotech companies

The Foundation operates with four key strategies:

Building alliances with academic and industry collaborators
Developing laboratory and animal research tools for researchers
Funding novel preclinical and clinical research in SMA
Educating legislators, government agencies, and media about SMA research

What They Don't Fund

Unsolicited grant applications: Only accepts applications by invitation following letter of intent review
Early-stage basic research: External funding limited to late translational work
Non-SMA research: Focused exclusively on spinal muscular atrophy
General operating support: Funding directed specifically to research projects
Patient support services: Focus is on therapeutic development, not direct patient care

Governance and Leadership

Executive Leadership

Loren A. Eng | President Loren co-founded the SMA Foundation in 2003 and has led it to invest over $150 million in research. She brings experience in investment banking, merchant banking, and media business development. She serves on boards including Columbia University Medical Center and Harvard University Stem Cell Institute. Education: BA (Wellesley College); MBA and MA in Education (Stanford University).

Karen S. Chen, PhD | Chief Executive Officer Dr. Chen oversees scientific and drug discovery programs with 35+ years of research management experience, including 15 years in pharmaceutical preclinical research. She previously served as Director at Roche Palo Alto (Neurosciences) and Elan Pharmaceuticals. Education: AB (Harvard); PhD in Neurosciences (UC San Diego).

Omar Khwaja, MD PhD | Chief Medical Officer A pediatric neurologist and geneticist with 20+ years in rare neurological disorders, Dr. Khwaja currently serves as Chief Medical Officer at Voyager Therapeutics and previously was Global Head of Rare Diseases at Roche. Education: University of Cambridge (medicine and PhD in molecular genetics).

Board of Directors

Dinakar Singh | Chairman of the Board Co-founder of the SMA Foundation and Founding Partner of TPG-Axon Capital. Former Partner at Goldman Sachs for 14 years, where he co-headed the Principal Strategies Department. Serves on Yale University Investment Committee and boards of NY Public Library, Columbia University Medical Center, The Rockefeller University, and Cold Spring Harbor Laboratories.

Douglas G. Cole, MD | Board Member General Partner at Flagship Ventures and co-founder of Ensemble Discovery. Former Instructor in Neurology at Harvard Medical School with post-graduate training at Johns Hopkins and Massachusetts General Hospital.

Gerald Fischbach, MD | Board Member Chief Scientist at the Simons Foundation, former NINDS Director, and past President of Society for Neuroscience. Member of the National Academy of Sciences.

Story Landis, PhD | Board Member Former NINDS Director (2003-2014), overseeing an annual budget of $1.6 billion supporting neuroscience research. Elected fellow of Institute of Medicine and American Association for the Advancement of Science.

Jonathan S. Leff | Board Member Partner at Deerfield Management and Chairman of Deerfield Institute. Former Warburg Pincus executive leading biotech/pharma investments.

Scientific Advisory Leadership

Marc Tessier-Lavigne, PhD | Chairman of the Scientific Advisory Board President of Stanford University and former President of Rockefeller University, with expertise in brain development and neuronal circuit formation.

Lee Rubin, PhD | Research Strategist Principal Investigator Professor at Harvard University's Stem Cell and Regenerative Biology department, with expertise in cell-based assays and drug discovery.

Key Quote from Leadership

"Since its inception, the Foundation has funded over $150 million in research" - Loren A. Eng, President

Application Process & Timeline

How to Apply

Important: The SMA Foundation operates an invitation-only grant application process. Unsolicited full applications are not accepted.

Step 1: Letter of Intent

Only principal investigators may submit
Send letter of intent to: research@smafoundation.org
Should outline research objectives, approach, and potential impact on SMA treatment development
Foundation reviews and selects projects aligned with strategic priorities

Step 2: Invitation to Apply

Foundation reviews letters of intent
Selected investigators receive invitation to submit full grant application
Full application guidelines provided with invitation

Step 3: Full Application

Submit detailed proposal as directed in invitation
Include specific budget, timeline, and deliverables

Decision Timeline

Letter of intent review timeline not publicly specified
Full application review and decision timeline varies by project scope
Given the focus on strategic partnerships, expect multiple rounds of discussion
Major partnership announcements suggest 3-6 month timeline from initial contact to award

Success Rates

Success rate data is not publicly disclosed. The invitation-only process means that only researchers whose letters of intent align with Foundation priorities are invited to submit full applications, likely resulting in higher success rates than open grant programs but with more stringent initial screening.

Reapplication Policy

The Foundation's reapplication policy is not explicitly stated. Given the invitation-only model:

Researchers whose letters of intent are not selected can submit new letters for different projects
No specified waiting period mentioned
Foundation encourages ongoing dialogue with research community
Changes in research focus or new developments may warrant resubmission

Application Success Factors

Strategic Alignment Requirements

The Foundation explicitly states that external funding is "limited in scope to work that will have immediate impact on late translational research or clinical trials." This is the primary filter for all funding decisions.

What They Look For:

Translational Readiness: Research must be advanced beyond basic discovery; ready to move toward clinical application
Clear Path to Clinical Trials: Projects should demonstrate how findings will directly inform or enable clinical testing
Novel Therapeutic Approaches: Innovative strategies for SMA treatment or disease modification
Validated Methodologies: Use of established research tools and models or development of new validated resources
Collaboration Potential: Opportunities to build partnerships between academic and industry researchers

Recent Funded Projects as Examples

Columbia University Center for Motor Neuron Biology and Disease ($15M): Comprehensive program to accelerate medical advances for SMA through motor neuron research
PTC Therapeutics Partnership ($8.5M): Small molecule development collaboration for drug discovery
Regeneron Pharmaceuticals ($1.65M): Development of new disease models to advance preclinical research

These examples show the Foundation funds:

Large-scale research centers with comprehensive SMA programs
Industry partnerships for therapeutic development
Tool and resource development that benefits the broader research community

Language and Terminology

When approaching the Foundation, use terminology that demonstrates:

Understanding of the translational research pipeline
Familiarity with SMA disease mechanisms and current therapeutic landscape
Focus on immediate impact and clinical applicability
Recognition of the Foundation's unique venture capital/biotech hybrid model
Awareness of research tools and resources the Foundation has already developed

Tips for Standing Out

Demonstrate Translational Readiness: Clearly articulate how your research moves beyond basic science toward clinical application
Show Industry Connections: Partnerships with pharmaceutical or biotech companies strengthen applications
Leverage Foundation Resources: Reference how you'll use or build upon SMA research tools the Foundation has already developed
Quantify Potential Impact: Specify how research will accelerate treatment development timeline
Highlight Team Expertise: Emphasize team members' track records in translating research to clinical trials
Address Regulatory Path: Discuss FDA considerations and clinical development strategy

Common Pitfalls to Avoid

Proposing early-stage basic research without clear translational pathway
Failing to demonstrate knowledge of existing SMA research landscape
Unclear connection to clinical trial development
Lack of industry collaboration or commercialization strategy
Proposals that duplicate existing Foundation-funded work
Insufficient evidence of research team's capacity to execute

Key Takeaways for Grant Writers

Invitation-Only Process: Do not submit unsolicited full applications. Start with a compelling letter of intent to research@smafoundation.org demonstrating how your research fits the Foundation's strategic focus on late translational work and clinical trials.
Translational Focus is Critical: The Foundation explicitly limits external funding to "work that will have immediate impact on late translational research or clinical trials." Early-stage basic research, no matter how innovative, is not aligned with current priorities.
Think Big and Strategic: Recent awards range from $1.65M to $15M, indicating the Foundation makes substantial investments in projects with transformative potential. Don't undershoot on ambition or budget if your project warrants it.
Leverage the Venture Capital Model: The Foundation operates as a "unique blend of non-profit, venture capital, and biotech entities." Approach them as you would a strategic investor—emphasize ROI in terms of treatment development acceleration, not just scientific discovery.
Industry Partnerships Strengthen Applications: The Foundation's major grants often involve academic-industry collaborations (e.g., Columbia, PTC Therapeutics, Regeneron). If you can demonstrate existing or potential industry partnerships, this aligns with their collaborative model.
Board Composition Signals Priorities: The board includes leaders from venture capital, pharmaceutical companies, and major research institutions. Applications should speak to both scientific excellence and business/commercialization strategy.
Resource Development Matters: Beyond funding individual research projects, the Foundation invests in tools, models, and resources that benefit the entire SMA research community. Projects that create shared resources may be particularly attractive.

References

SMA Foundation Official Website - About Us https://smafoundation.org/about-us/ Accessed: November 13, 2025
SMA Foundation - Research Funding https://smafoundation.org/research/funding/ Accessed: November 13, 2025
SMA Foundation - Board of Directors https://smafoundation.org/about-us/board/ Accessed: November 13, 2025
SMA Foundation - Team https://smafoundation.org/about-us/team/ Accessed: November 13, 2025
SMA Foundation - Contact Us https://smafoundation.org/about-us/contact-us/ Accessed: November 13, 2025
ProPublica Nonprofit Explorer - Spinal Muscular Atrophy Foundation https://projects.propublica.org/nonprofits/organizations/10759380 Accessed: November 13, 2025 (Financial data from 2023 Form 990)
Columbia University Irving Medical Center - "Columbia Receives Up To $15 Million From Spinal Muscular Atrophy Foundation" https://www.cuimc.columbia.edu/news/columbia-receives-15-million-spinal-muscular-atrophy-foundation Accessed: November 13, 2025
SMA Foundation Press Release - "PTC Therapeutics and Spinal Muscular Atrophy Foundation Announce $8.5 million Small Molecule Development Collaboration" https://smafoundation.org/foundation-updates/ptc-therapeutics-and-spinal-muscular-atrophy-foundation-announce-8-5-million-small-molecule-development-collaboration/ Accessed: November 13, 2025
Grantmakers.io - Spinal Muscular Atrophy Foundation Profile https://www.grantmakers.io/profiles/v0/010759380-spinal-muscular-atrophy-foundation/ Accessed: November 13, 2025
National Organization for Rare Disorders - Spinal Muscular Atrophy Foundation https://rarediseases.org/organizations/spinal-muscular-atrophy-foundation/ Accessed: November 13, 2025