named programs · 2 · from sources
What they call their work
Daybreak Children's Rare Disease Fund
Primary initiative focused on raising awareness, supporting research, and promoting timely development of diagnostics and treatments for rare genetic diseases in children
Online Information and Education Platform
Provides general information about rare genetic diseases through its website, including content for public education and awareness, not intended as medical advice
activities · 6 groups
What they do
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Rare Genetic Disease Research and Advocacy 3 activities
- Conducting awareness and advocacy campaigns for rare genetic diseasesConducts awareness-raising activities and advocacy campaigns about rare genetic diseases, promoting the development and availability of safe and effective diagnostics and treatments. Advocates for timely access to therapies and regulatory advancements.
- Providing direct treatment access for rare disease patientsProvided gene therapy treatment to U.S. patient Sebastian with ADA-SCID, resulting in sustained recovery and improved health, demonstrating direct clinical intervention for rare genetic disease patients.
- Securing regulatory approval for gene therapiesSecured global regulatory approval for Strimvelis, the first ex-vivo stem cell gene therapy for children with ADA-SCID, marking the first corrective gene therapy for children approved worldwide.
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Cystic Fibrosis Research and Support Funding 1 activity
- Conducting professional fundraising campaignsOperates a team of professional fundraisers in San Diego, California, conducting face-to-face outreach in metropolitan areas of California to enroll regular donors and raise funds for scientific research on rare genetic diseases.
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Genetic and Neurological Disease Research Funding 1 activity
- Funding research for cures of rare genetic diseasesFunds research for cures of rare genetic diseases by directing resources to Fondazione Telethon Italia and its research centers, TIGEM and SR-TIGET. Supported 2,629 research projects and activities related to rare genetic diseases.
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Organization Website and Online Platform Management 1 activity
- Operating online platforms for rare disease information and community engagementOperates online platforms including message boards, blogs, chatrooms, and forums to support engagement around rare diseases. Provides free access to websites and online services related to rare diseases and shares third-party articles and information for public benefit.
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Charity Event Organization 1 activity
- Organizing fundraising events and campaignsRaised approximately $39 million through a fundraising marathon via telephone and online donations, sale of heart-shaped chocolates at volunteer-run stalls, and partner company events. Hosted musical performances and auctioned high-value items such as the first Ferrari 458 Speciale A sold in the U.S., generating $900,000 in net proceeds.
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Biomedical Research and Innovation 1 activity
- Supporting clinical research and gene therapy developmentConducts and supports clinical research in gene therapy, including successful trials for Wiskott-Aldrich syndrome and metachromatic leukodystrophy, with results published in Science. Conducted research on gene silencing via epigenetic modifications published in Cell, and synthesized artificial proteins to induce targeted gene silencing.
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financials · form 990 · fy2020
revenue
Total revenue$9K
Contributions & grants$9K100%
Program service revenue$00%
Investment income$00%
Other revenue$0
expenses
Total expenses$15K
Program expenses0%
Admin / overhead100%
Fundraising0%
Salaries & benefits$0
Grants paid out$0
Largest expense lineProfessional Fees
balance sheet
Total assets$80K
Cash$80K
Investments$0
Liabilities$2K
Net assets$78K
Liquid reserves62.4 mo
1 years on record · 2020–2020
leadership · form 990 part vii · fy2020
Who runs it
board members · 7
- CHRISTIAN MORETTI — SECRETARY
- DANIELE BODINI — DIRECTOR
- FRANCESCA PASINELLI — VICE CHAIR
- LUCA DI MONTEZEMOLO — CHAIR
- MICHAEL CAPLAN — DIRECTOR
- PRIYA STEPHEN — DIRECTOR
- TIZIANA CIRACO — TREASURER
relationships · 26
Who they work with
- Baylor College of Medicine Partner — Scientific collaboration and joint research endeavors with TIGEM, including shared personnel and research initiatives.
- Daybreak Partner — Received net proceeds from the auction of the Ferrari 458 Speciale A.
- Fondazione Telethon Partner — Partnered in the development and advancement of gene therapy for rare diseases, including the approval of Strimvelis.
- Fondazione Telethon Italia Partner — Primary beneficiary of Daybreak's fundraising and awareness efforts.
- Fondazione Telethon Italia Partner — Primary beneficiary of funding and collaborative efforts for rare disease research.
- Fondazione Telethon Italia Partner — Primary beneficiary of funding and support efforts.
- Fondazione Telethon Italia Partner — Primary beneficiary of fundraising and support efforts.
- Fondazione Telethon Italia Partner — Primary beneficiary of research funding and support.
- Friends of Telethon Foundation Italy Network — Operates as dba Daybreak Children’s Rare Disease Fund under Friends of Telethon Foundation Italy
- Friends of the Telethon Foundation Italy, Inc. Network — Operating name "Daybreak Children’s Rare Disease Fund" is used by Friends of the Telethon Foundation Italy, Inc.
- GSK Partner — Collaborated on the development and approval of Strimvelis for ADA-SCID through a strategic partnership.
- Ospedale San Raffaele Partner — Collaborated with GSK and Fondazione Telethon on the development of gene therapies, including Strimvelis.
- RAI Partner — Partnered to broadcast the fundraising campaign on national TV and radio networks.
- SR-TIGET Partner — Collaborates with SR-TIGET to provide gene therapy treatments for children with rare diseases.
- SR-TIGET Partner — Research center supported through funding and advocacy.
- SR-TIGET Partner — World-class research center receiving support for rare disease research.
- SR-TIGET Partner — World-class research center supported as a primary beneficiary of the organization's efforts.
- SR-TIGET Partner — World-class research center supported by Daybreak through Fondazione Telethon Italia.
- SR-Tiget Partner — Research center supported through funding.
- San Raffaele Telethon Institute for Gene Therapy Partner — Collaborates on gene therapy research
- TIGEM Partner — Research center supported through funding and advocacy.
- TIGEM Partner — World-class research center receiving support for rare disease research.
- TIGEM Partner — World-class research center supported as a primary beneficiary of the organization's efforts.
- TIGEM Partner — World-class research center supported by Daybreak through Fondazione Telethon Italia.
- Telethon Italia Network — Founded by Susanna Agnelli, mother of honorary board member Priscilla Rattazzi.
- Tigem Partner — Research center supported through funding.
strategies · 3
How they approach the work
Named approaches extracted from this org’s sources. Where others share an approach, follow it to see the full set of orgs running it.
- Gene Therapy as Curative Interventionmethodology: gene_therapyBy developing ex-vivo stem cell gene therapies and leveraging gene silencing techniques, we correct genetic defects at the cellular level to produce durable cures for severe congenital immunodeficiencies, because these approaches address root causes rather than symptoms.
- Merit-Based Peer Review for Research Selectionmethodology: peer_reviewBy using a peer-reviewed, merit-based selection process, we ensure that only the highest-potential research initiatives receive funding, because scientific evaluation by experts increases the likelihood of clinical impact and efficient use of resources.
- Strategic Research Institution Partnershipsmethodology: strategic_research_partnershipBy directing research funding to established, high-capacity institutions with proven expertise in rare genetic diseases, we accelerate therapeutic development and maximize impact, because these institutions have the infrastructure, credibility, and scientific rigor to advance cures efficiently.