ATLANTA (October 24, 2023) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a project at Armatus Bio that will advance a novel miRNA gene therapy candidate for CMT type 1A designed to reduce PMP22 overexpression in Schwann cells, a known genetic driver of the disease. The treatment would be delivered via a single intrathecal injection, with the objective of achieving clinically meaningful functional improvements, enabling patients to retain activities of daily living. If successful, Armatus aims to initiate clinical trials of ARM-101 in early 2025.
“Armatus Bio is advancing a gene therapy candidate that has shown promising results in early in vitro and animal model studies,” says Cleary Simpson, CEO of CMTRF. “In order to progress into human clinical trials, it is important to demonstrate that the therapy can reach specific targets in animal models more closely related to humans. Foundational studies like these enable promising scientific concepts to move into human testing with increased confidence in safety and efficacy for a population in need.”
“We are conducting a study of the ARM-101 biodistribution to confirm the Schwann cell targeting success seen in mice and verify appropriate human doses in a model that more closely correlates to human biology,” says Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “These foundational data are not only critical to advancing ARM-101 development, but also provide crucial information about the ability of precision therapies to target appropriate tissues implicated in neuropathies.”
Armatus Bio, based in Columbus Ohio, is an emerging biotechnology company applying the rigor of advanced bioengineering to the complex challenge of genetic medicine. Through dynamic collaborations with renowned gene therapy experts in Ohio, the company is building a pipeline of novel therapeutic candidates designed to overcome the limitations of today’s approaches and propel the next generation of genetic medicines.
CMT Research Foundation (CMTRF) is a patient-led, non-profit focused on delivering treatments and cures for CMT. The foundation identifies significant obstacles or deficiencies impeding progress towards a cure and seeks out collaborators to address these issues. To date, CMTRF has funded 19 projects, of which 6 are completed. Of those 6 completed projects, 5 have clinical candidates. CMTRF’s mission to invest in promising science with high potential of leading to treatments and cures was proven effective and ground-breaking when DTx Pharma with a CMTRF- backed program as its lead candidate was acquired by Novartis for $1 billion. Founded by two patients who are driven to expedite drug delivery to people who live with CMT, the 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.
*Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms, causing walking, running and balance problems as well as abnormal functioning of hands and feet. CMT affects one in 2,500 people (about the same prevalence as cystic fibrosis), including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT.