CAMBRIDGE, Mass. — CREATE Medicines has received approval in Australia to begin a first-in-human Phase 1/2 clinical trial of CRT-402, an in vivo CAR-T therapy being developed for autoimmune diseases.
The approval from Australia’s Human Research Ethics Committee clears CREATE to begin enrolling patients with systemic lupus erythematosus, systemic sclerosis and idiopathic inflammatory myopathies.
The study represents CREATE’s first clinical program outside oncology and the fourth candidate from its immune-programming platform to enter clinical testing.
CRT-402 is a CD19-targeted therapy designed to reprogram a patient’s T cells directly inside the body to eliminate autoreactive B cells and potentially drive autoimmune disease into remission.
Unlike conventional CAR-T therapies, CRT-402 does not require a patient’s cells to be removed, modified in a laboratory and reinfused. CREATE said the in vivo approach could simplify manufacturing and make CAR-T treatment available to more patients.
“Our clinical experience in oncology has demonstrated the potential of CREATE’s platform to deliver in vivo CAR therapies directly in patients, and CRT-402 represents a natural extension of that platform into autoimmune disease,” said Daniel Getts, Ph.D., Chief Executive Officer and Co-Founder of CREATE Medicines.
Getts said advancing the candidate into clinical testing supports the potential use of the company’s platform across multiple therapeutic areas.
“Patients with B cell-driven autoimmune diseases, including myositis, urgently need therapies that can deliver deep and durable remission,” said Merrilee Needham, Foundation Chair in Neurology at Fiona Stanley Hospital and Principal Investigator for the trial.
Needham said CRT-402’s potential to achieve deep B-cell depletion without conventional cell manufacturing could allow more patients to receive treatment.
CRT-402 is delivered using CREATE’s proprietary messenger RNA-lipid nanoparticle platform. The therapy is designed to provide deep B-cell depletion and an immune reset while offering the potential for repeat dosing and off-the-shelf administration.
In preclinical studies, CRT-402 produced deep and durable B-cell depletion in nonhuman primates.
