Vita Raises $32M to Advance Cell Therapies for LGMD, Other Dystrophies

Vita Therapeutics announced that it has raised $32 million in financing to advance its work into cell-based therapies for muscular dystrophy.

“At Vita Therapeutics our mission is to deliver long-term disease-modifying cell engineered treatments for patients living with muscular dystrophies and other high unmet medical needs,” Douglas Falk, the company’s CEO, said in a press release.

Money raised, which came through a Series financing round, “will enable the company to take the next steps toward achieving our mission,” he added.

The funding will support further preclinical studies of VTA-100, Vita’s lead treatment candidate for limb-girdle muscular dystrophy (LGMD) type 2A/R1, the most common form of the disease. The company is planning to request U.S. Food and Drug Administration (FDA) approval to initiate clinical trials of VTA-100.

VTA-100 is designed to be an autologous treatment that combines gene correction and stem cell technology to help repair and replace muscle cells, Vita reports. The treatment, in essence, involves collecting cells from a patient, then using genetic and biochemical engineering to correct the disease-causing genetic defect in cells, and to prompt the cells to grow into working muscle cells when administered to a patient.

Vita is also developing VTA-200, which similarly aims to use genetically engineered stem cells. However, whereas VTA-100 is autologous — using cells from the patient being treated — VTA-200 is designed to be hypoimmunogenic (universal), meaning that cells from one donor could be used to treat various patients.

VTA-200 is intended to treat multiple types of muscular dystrophy, Vita reports. A third treatment candidate, VTA-300, uses an undisclosed cell type.

The funding will also be used to further develop VTA-200 and VTA-300, as well as to help manufacture cells needed for clinical evaluation, and to offset clinical trial costs for testing VTA-100.

“Cell therapies have two grand challenges — getting enough cells and differentiating them into the right cell type to make a long-term impact on a patient’s disease,” said James Peyer, PhD, recently appointed as a board member at Vita and CEO of Cambrian Biopharma, which led the $32 million financing effort.

“By mastering the transition from [stem cell] to muscle stem cell, Vita can make an unlimited amount of carefully defined muscle stem cells, which has never been possible before,” Peyer added. “I have no doubt Vita will become a genre-defining cell therapy company.”

Vita is an affiliate of Cambrian Biopharma, and its treatment approach stems from work that began at labs at Johns Hopkins University and the Kennedy Krieger Institute.