Chondrial Therapeutics is an emerging biotechnology company focused on the treatment of rare mitochondrial diseases. Mitochondrial disorders are common and are very difficult to treat. Our mission is to develop therapies for these diseases. We are initially targeting FA. FA is the most common ataxia in men and is caused by a deficiency of a key protein, frataxin, which is normally present in the mitochondria of all tissues.
MILESTONES / INNOVATIONS
Dr. Mark Payne, the discoverer of Chondrial’s lead drug candidate, CTI-1601, began investigating approaches to deliver the deficient protein, frataxin, to mitochondria in cells when he was a researcher at Wake Forest University. In 2016, Chondrial Therapeutics, Inc. was established with funding of $22.6 million primarily from Deerfield Management. This enabled the company to hire a management team, establish a basic science laboratory in Philadelphia to advance the understanding of FA and the effects of frataxin, and begin the manufacturing development program to support a Phase 1 clinical trial for CTI-1601.
In designing the Phase 1 trial, Chondrial is obtaining input from patients and caregivers of patients with FA. Chondrial recognizes the importance of reaching out to patients and caregivers to ensure they play an active and critical role in advancing drug development.
“We are excited to work with patients and caregivers on this project because of their deep understanding of the disease and the patients impacted by it.”
– Dr. Carole Ben-Maimon ,
ADVICE:
“I believe that Chondrial is in a prime position to advance a potentially groundbreaking therapeutic for patients with Friedreich’s Ataxia and capitalize on a broader vision of targeting and treating rare, debilitating mitochondrial diseases,” says Dr. Carole Ben-Maimon, President and CEO, Chondrial Therapeutics, Inc. “We are excited to work with patients and caregivers on this project because of their deep understanding of the disease and the patients impacted by it”.
WHY PHILLY:
Chondrial’s research and development is in a newly established laboratory in University City, near the University of Pennsylvania where there is easy access to all the key stakeholders. The Children’s Hospital of Philadelphia is nearby and is home to the Friedreich Ataxia Center of Excellence, where more than 500 individuals with FA have been diagnosed and receive ongoing care. It’s a short train ride to the Food & Drug Administration, the regulatory agency with oversight for all new drugs, and to the investor community which has provided the necessary capital.
