UMDF Announces Inaugural Venture Philanthropy Investment: The Mito Fund Invests $500k in Pierrepont Therapeutics, Inc. to Help Move a Mitochondrial Disease Therapy Forward

(APRIL 4, 2024) The United Mitochondrial Disease Foundation (UMDF) today announced the inaugural investment for its venture philanthropy initiative, The Mito Fund.

The Mito Fund has invested $500,000 in Pierrepont Therapeutics, Inc., a company focusing on treatments for primary mitochondrial and rare diseases, for the development of PTI-501, a proprietary intracellular enzyme replacement therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE).

“We’ve said all along that time is something mitochondrial disease families just don’t have, especially when it comes to something as devastating as MNGIE,” said UMDF President & CEO Brian Harman. “This investment could be truly transformational for our community. We’re excited to be partnering with Pierrepont to advance this potential therapy.”

Following an extensive review process, the Pierrepont investment was recommended by UMDF’s Venture Philanthropy Investment Committee, an advisory group consisting of world-class experts in the areas of mitochondrial disease, pharmaceutical development, and venture investments. The committee’s recommendation was unanimously approved by UMDF’s Board of Trustees.

“Pierrepont is honored to receive the first investment from The Mito Fund, and we look forward to continuing our collaboration with the UMDF,” said Daniel DiPietro, co-founder of Pierrepont.  “PTI-501 is a targeted therapeutic with a known biomarker, and we are eager to advance this therapy into clinical development to help MNGIE patients and families worldwide- this investment will help us achieve that goal.”

MNGIE is a rare mitochondrial disease that primarily impacts the digestive and nervous systems. While the age of onset varies, the disease is relentlessly progressive and causes severe malnutrition and gastrointestinal complications that typically lead to death.

“I firmly believe this treatment approach holds great potential for MNGIE patients,” said Michio Hirano, MD, of Columbia University. “MNGIE is a disease with a defined natural history, which makes it ripe for a targeted therapy. From a patient perspective, this is a disease that is debilitating and often fatal with limited therapeutic options, such as allogeneic stem cell or liver transplant, that pose significant risk.”

Launched in the summer of 2023, The Mito Fund is UMDF’s most ambitious effort to date to ramp up the development of mitochondrial disease treatments and cures. The fund’s venture philanthropy model is designed not only to financially support the development of the next generation of mitochondrial disease treatments, but also to leverage UMDF’s experience and network to accelerate the development process and lower the risk associated with entry into the field for a wide range of investors and researchers.

“Seeing venture philanthropy come to fruition at UMDF after a decade of consideration is incredibly exciting,” said Dr. Philip Yeske, UMDF Science and Alliance Officer. “The time was finally right to create and launch such an initiative for the mitochondrial disease community and we look forward to continuing the evaluation of investment opportunities at many more companies as part of the UMDF research mission.”

Companies exploring the mitochondrial science space are encouraged to start a conversation about investment opportunities at Proposals are accepted on a rolling basis.

UMDF thanks the Kallaos and Wright families for their founding gifts that helped make the launch of The Mito Fund possible. If you’d like to join them as a donor to The Mito Fund, visit, email, or call 1-888-317-8633 to learn more.

About The United Mitochondrial Disease Foundation
For more than 25 years, The United Mitochondrial Disease Foundation (UMDF) has built a global network of patients, researchers, clinicians, institutions, and industry partners dedicated to fighting mitochondrial disease. Together with the mito community, UMDF is committed to a mission of funding the best science no matter where it is found in the world while at the same time providing critical education, advocacy, and support-focused programs and services to patient families. For more information, visit

In the last year …  

  • Nearly 4,000 patients and caregivers attended UMDF support meetings.
  • More than 800 clinicians listened in on UMDF’s Bench-to-Bedside seminars.
  • UMDF advocates had 1,600 touchpoints with their congressional offices.
  • UMDF provided almost 600 patients with no-cost genetic testing.
  • More than $1.5 million invested in mitochondrial disease research efforts.

About Pierrepont Therapeutics, Inc.  
Pierrepont Therapeutics, Inc. is dedicated to developing targeted treatments for primary mitochondrial and other rare genetic diseases. Pierrepont’s lead product candidate, PTI-501 (formerly ENTR-501) is a proprietary intracellular enzyme replacement therapy for the treatment of MNGIE that is exclusively licensed from Entrada Therapeutics, Inc. (Nasdaq: TRDA). The founders of Pierrepont have unique expertise and success in mitochondrial disease drug development, and have strong relationships with KOLS, families, and patient advocacy groups in the mitochondrial disease community.  For more information, please visit or contact

About Venture Philanthropy
Venture philanthropy donors make a charitable contribution to a restricted philanthropic fund housed within a recognized 501(c)(3) organization like UMDF. Funds are then invested by the organization in promising for-profit companies similar to venture capital, but with the critical difference that venture philanthropist donors do not individually benefit from any financial returns on successful investments. Instead, those returns are funneled directly back to the philanthropic fund to be reinvested in new ventures.