Mila's Miracle Foundation

What Mila's Miracle Foundation does

Mila's Miracle Foundation is a 501(c)(3) nonprofit organization founded in December 2016 by Julia Vitarello after her daughter Mila was diagnosed with Batten disease, a fatal neurodegenerative condition. The foundation is headquartered in Boulder, Colorado, and operates with a small team under Vitarello's leadership as Founder and CEO. Its mission is to make individualized medicines accessible to children with rare genetic diseases, and it pursues that mission by funding research, convening clinical partners, and building advocacy infrastructure for families affected by rare conditions. The foundation runs on a donation-based revenue model, with all gifts being tax-deductible, and supplements individual donations with grants from institutional funders such as the Chan Zuckerberg Initiative.

The foundation's defining technological achievement is Milasen, the world's first drug customized for a single patient—an antisense oligonucleotide (ASO) therapy developed in partnership with Dr. Timothy Yu at Boston Children's Hospital to target Mila's specific CLN7 Batten disease mutation, which received FDA approval in December 2019. The foundation's portfolio extends across multiple programs: a CLN7 gene replacement therapy clinical trial with Dr. Steven Gray and UT Southwestern; the Little Legacies brain donation program with the NIH NeuroBioBank and Brain Donor Project; the Cell Atlas Project for single-cell mapping of pediatric genetic disease; the Batten and Neurodegenerative Disease Clinic at Children's Hospital Colorado; and the UK Rare Therapies Launch Pad pilot with MHRA, Genomics England, and Oxford University to establish regulatory pathways for individualized treatments. The foundation reaches a global community of over 6,000 supporters across 52 countries through Mila's Tribe, which drives grassroots fundraising via GoFundMe, community events, and earned media.

The foundation's go-to-market motion is community-led, relying on grassroots fundraising, social media engagement, direct donation appeals, and high-profile media coverage in publications such as The New York Times, BBC, The Economist, STAT, and The New England Journal of Medicine. Rather than developing drugs in-house, the foundation operates as a convener and funder, directing capital to partner institutions and researchers. Its customer base consists of rare disease families seeking hope and treatment access, supported by a donor network of individuals, corporations, and institutional grant providers. The foundation's founder has also co-founded EveryONE Medicines and the N=1 Collaborative, signaling a broader strategic ambition to scale individualized medicine beyond the Batten disease community.

Name

Mila's Miracle Foundation

Legal name

Mila's Miracle Foundation, Inc.

Website

https://milasmiracle.org

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

1–10 employees

Short description

Mila's Miracle Foundation is a 501(c)(3) nonprofit that pioneered Milasen, the first personalized antisense oligonucleotide drug for a single patient, and funds individualized medicines for children with rare genetic diseases through research partnerships, clinical programs, and the UK Rare Therapies Launch Pad.

Ownership category

akta.pro rank

Product category

Nonprofit Rare Disease Research & Advocacy

NAICS

Voluntary Health Organizations (813212), Grantmaking and Giving Services (8132), Social Assistance (624), Other Individual and Family Services (624190)

SIC

Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731), Services-Social Services (8300)

akta.pro primary industry

Rare Disease & Special Needs Support Organizations (BPAGACAM)

akta.pro secondary industries

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Health & Medical Research Grantmaking Foundations (BPAGAKAL)

Keywords

• Rare disease research
• Individualized medicine advocacy
• Antisense oligonucleotide therapy
• Nonprofit medical foundation
• Genetic disease treatment

Headquarters

HQ city

Boulder

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

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

Revenue model

1. Donations and Fundraising: As a 501(c)(3) nonprofit organization, Mila's Miracle Foundation generates revenue through charitable donations from individuals, corporations, and events. The foundation runs GoFundMe campaigns and organizes community fundraising events. All donations are tax-deductible.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels7 records

Core offering

Mila's Miracle Foundation is a 501(c)(3) nonprofit that develops, funds, and advocates for individualized medicines for children with rare genetic diseases such as Batten disease. It pioneered Milasen, the first drug customized for a single patient, and now operates a portfolio that includes a CLN7 gene replacement therapy clinical trial, a specialized multidisciplinary clinic at Children's Hospital Colorado, a brain donation program (Little Legacies) with an associated Cell Atlas Project, and regulatory/reimbursement pilot initiatives such as the UK Rare Therapies Launch Pad.

Product overview

Mila's Miracle Foundation operates a portfolio of programs centered on individualized medicines for rare genetic diseases. The core offering is Milasen, the world's first personalized antisense oligonucleotide (ASO) treatment designed for one person. The foundation's work spans clinical care (Batten & Neurodegenerative Disease Clinic), research infrastructure (Cell Atlas Project, CLN7 Gene Replacement Therapy), family support (Little Legacies brain donation program), and systemic change initiatives (Mila to Millions, UK Rare Therapies Launch Pad). The foundation also operates advocacy tools including the 'This Is Today' photo journal to influence policy decisions. The foundation's products work together to advance individualized medicine from research through clinical access.

Differentiator

Problem solved

Functional benefit

Brands

• Milasen: The first-ever drug customized for a single patient, an antisense oligonucleotide (ASO) treatment for Mila's specific CLN7 Batten Disease mutation.
• Mila to Millions
• Little Legacies

Products and services

• Milasen (Individualized Antisense Oligonucleotide Treatment) The world's first drug customized for a single patient. Milasen is an antisense oligonucleotide (ASO) therapy that targets the individual patient's specific disease-causing mutation, originally developed for Mila Makovec's CLN7 form of Batten disease and pioneered through a partnership with Dr. Timothy Yu at Boston Children's Hospital.
• CLN7 Gene Replacement Therapy A novel gene therapy that delivers a working copy of the MFSD8/CLN7 gene via a one-time injection into the cerebrospinal fluid, intended for children with Mila's form of Batten disease (CLN7). Developed in partnership with Dr. Steven Gray, UT Southwestern Medical Center, Aashi's Hope, and Batten Hope.
• Batten & Neurodegenerative Disease Clinic A specialized multidisciplinary clinic at Children's Hospital Colorado offering families affected by Batten and other neurodegenerative diseases access to neurology, special care, rehabilitation medicine, neuropsychology, and physical/occupational/speech therapies in one location.
• Little Legacies Program A program supporting families of children with genetic diseases through the emotional decision of brain/tissue donation for research, partnering with NIH NeuroBioBank, Brain Donor Project, and University of Maryland to coordinate collection, storage, and sharing of donations, and producing the Cell Atlas single-cell map for pediatric genetic disease.

Quantifiable outcome

• First person in the world to receive a personalized medicine (Milasen)
• +1 more outcomes

Named customers1 record

Segments1 record

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

Twelve partnerships are on record, tiered flagship, core and minor.

• UK Rare Therapies Launch PadflagshipStrategic or Co-development Partner · 1 January 2023Groundbreaking pilot endorsed by the UK government to prove a new scalable, sustainable model for access to individualized treatments, shifting the regulatory and reimbursement pathways. Includes partnership with MHRA, Genomics England, and Oxford University.
• NIH NeuroBioBankcoreStrategic or Co-development Partner · 1 January 2020Partnership with the NIH NeuroBioBank for coordinating collection, storage and sharing of tissue donations from children with genetic diseases. Part of the Little Legacies program to help families navigate brain donation and make donations available to researchers worldwide.
• Brain Donor ProjectcoreStrategic or Co-development Partner · 1 January 2020Partnership to help families coordinate tissue donations for research. Working together to create a supportive space for families to ask questions and navigate the process of gifting their child's brain to science.
• University of MarylandcoreStrategic or Co-development Partner · 1 January 2020Partner in the Little Legacies program working with an experienced and collaborative team to coordinate the collection, storage and sharing of tissue donations so they are easily available to dedicated researchers around the world.
• Children's Hospital ColoradocoreStrategic or Co-development Partner · 1 January 2020The foundation launched the Batten and Neurodegenerative Disease Clinic at Children's Hospital Colorado to provide families with children affected by neurodegenerative diseases access to specialized multidisciplinary care, all in one place. The clinic collects critical research data while providing care.
• Crispin Porter + BoguskyminorGTM or Marketing Partner · 1 January 2018World-renowned advertising agency that came into the family's house just weeks after Mila's diagnosis with a film crew and spent hundreds of hours editing the video that allowed the world to know Mila and be moved to help.
• GoFundMecoreGTM or Marketing Partner · 1 January 2018GoFundMe headquarters stepped up to help get the foundation's story out to the masses. The foundation runs multiple campaigns on the GoFundMe platform to raise critical funds.
• Dr. Steven GraycoreStrategic or Co-development Partner · 1 January 2017Partnership with renowned gene therapist Dr. Steven Gray to develop a novel gene therapy direct to the brain for children with CLN7 Batten Disease. This promising approach replaces the broken gene with the correct one in a one-time shot into the cerebrospinal fluid.
• UT SouthwesterncoreStrategic or Co-development Partner · 1 January 2017Partnership with UT Southwestern Medical Center for the CLN7 gene therapy clinical trial. The trial was developed in partnership and launched in Spring 2021.
• Aashi's HopecoreStrategic or Co-development Partner · 1 January 2017Partnership with Aashi's Hope (Beat Batten) to jointly develop and fund the CLN7 gene therapy clinical trial.
• Batten HopecoreStrategic or Co-development Partner · 1 January 2017Partnership with Batten Hope to jointly develop and fund the CLN7 gene therapy clinical trial.
• Boston Children's HospitalcoreStrategic or Co-development Partner · 1 January 2016The foundation partnered with Boston Children's Hospital to develop Milasen, the first-ever individualized medicine. Led by Dr. Timothy Yu at the Division of Genetics and Genomics, this antisense oligonucleotide treatment was developed specifically for Mila and has since opened up an entirely new field of individualized medicines. The partnership continues with ongoing research and the Cell Atlas Project.

Scale indicators5 records

Recent moves6 records

Expansion highlights6 records

Broad incumbents

• National Organization for Rare Disorders (NORD): Largest US rare disease advocacy umbrella organization; awarded Mila's Miracle Foundation the 2020 Rare Impact Award and operates overlapping advocacy, policy, and patient-support programs.
• Parent Project Muscular Dystrophy (PPMD): Established rare pediatric disease nonprofit that pioneered parent-driven research funding and regulatory advocacy for Duchenne; comparable in operating model and policy influence.
• Global Genes: Major rare disease patient advocacy organization that presented Mila's network with the Rising Star Award; comparable in awareness-building and rare-disease community programming.

Emerging players

• Cookies4Cures: Grassroots, community-led fundraising model (recognized via Global Genes Rising Star Award) that mirrors Mila's Miracle Foundation's early-stage GoFundMe-driven growth playbook for rare pediatric diseases.

Direct peers

• N=1 Collaborative: Co-founded by Julia Vitarello to address systemic barriers for individualized medicines; explicitly built on learnings from Milasen and operates in the same n-of-1 therapeutics advocacy space.
• Cure SMA: Patient-founded nonprofit that drove development and approval of multiple SMA therapies (including individualized antisense approaches); highly comparable model of rare pediatric disease family-led foundation.
• Beyond Batten Disease Foundation: Focused on juvenile Batten disease (CLN3) with similar mission of funding research and driving treatments; comparable nonprofit structure and disease-area focus.
• EveryONE Medicines: Founded by Julia Vitarello to develop individualized medicines at scale; direct continuation of the Milasen platform concept into a therapeutics-focused entity.
• Aashi's Hope: Active co-development partner on the CLN7 gene therapy trial; another small, family-led Batten disease foundation with directly overlapping program.
• Batten Hope: Co-development partner on the CLN7 gene therapy clinical trial with UT Southwestern; operates a near-identical family-driven Batten disease model.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/stopbatten
• LinkedInlinkedin.com/company/milas-miracle-foundation

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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Frequently asked questions about Mila's Miracle Foundation