The Mito Fund Makes First Investment in Pierrepont

UMDF Announces Inaugural Venture Philanthropy Investment

The Mito Fund Invests $500k in Pierrepont Therapeutics, Inc.
to Help Move a Mitochondrial Disease Therapy Forward

The United Mitochondrial Disease Foundation (UMDF) has 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 Enzyme replacement for Mitochondrial Neurogastrointestinal Encephalomyopathy (

“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. 

scroll to keep reading >>

On the heels of the first investment, UMDF President & CEO Brian Harman talks about The Mito Fund.

Why Pierrepont? UMDF Science & Alliance Officer Dr. Philip Yeske talks about The Mito Fund’s first investment.

“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 our “Spark a Conversation” page, email, or call 1-888-317-8633 to learn more.

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

Identifying & Researching

In the first two years, The Mito Fund will identify and invest in a portfolio of four to six companies, with an eye to double the initial portfolio size in the three year.

Calculating Risk vs Reward

The Mito Fund at UMDF is managed by the board-approved Venture Philanthropy Investment Committee (VPIC)

Funding The Best Science

Investments are focused on the most promising developments in i) medical devices, ii) small molecules (traditional drug development using novel chemical matter,) and iii) biologics (drugs such as gene and cell therapies made from complex molecules manufactured using living microorganisms, plants, or animal cells.)


Looking to support UMDF through Venture Philanthropy?