Despite the widespread freeze on clinical research due to the coronavirus pandemic, Bala Cynwyd-based Larimar Therapeutics is advancing clinical trials for Friedreich’s ataxia, a complex inherited rare disease that causes progressive nervous system damage and movement problems, writes Queen Muse for the Philadelphia Magazine.
The disease affects around one in 50,000 people and currently there is no cure or effective treatment for this debilitating ailment.
According to Carole Ben-Maimon, president and chief executive officer of Larimar Therapeutics, Friedreich’s ataxia is a uniquely challenging disease to treat because it is caused by a deficiency in the mitochondrial protein, frataxin.
“The protein works inside the mitochondria of the cell,” she said. “To treat it, you have to get the protein across the cell membrane and across the mitochondrial membrane, which is a challenge.”
The company launched a Phase I clinical trial to determine the safety and tolerability of CTI-1601 in November. This recombinant fusion protein is intended to deliver human frataxin to patients suffering from FA.
The trial was paused in March, but restarted in late July. Now the company expects to have topline results in the first half of 2021.
Read more about Larimar Therapeutics at the Philadelphia Magazine by clicking here.
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