Sigilon Therapeutics’s Afibromer SIG-003 candidate for the long-term treatment of patients with hemophilia B recently received an Advanced Therapy Medicinal Product (ATMP) designation in the European Union.
The designation was granted by the Committee for Advanced Therapies of the European Medicines Agency (EMA) and follows a recommendation granted by the committee earlier in November 2017, specifically regarding allogeneic cells (from a matched donor) genetically modified to express human factor IX protein embedded in the Afibromer biomaterial (SIG-003) for hemophilia B.
Afibromer Living Therapeutics are based on novel biomaterials discovered by Sigilon, called Afibromers. Sigilon’s proprietary cells are engineered to secrete therapeutic proteins which, in combination with its proprietary technology, allow levels of the missing protein in the body to be restored for long periods of time following a single intervention.
Sigilon’s afibrotic polymers are designed to avoid triggering unwanted immune responses, specifically fibrotic responses, which the body uses to isolate foreign bodies.
The Afibromer technology could have broad therapeutic applications, according to the company, as it will allow the delivery of cells without the need for immune system suppression.
Potential benefits of these new cell therapies include improved outcomes over current therapies, constant levels of protein, years of therapy provided without the need for re-dosing, and replacement of frequent injection/infusion, as well as the delivery of proteins for previously untreated diseases.
For hemophilia B patients, Afibromer SIG-003 would allow the delivery of the missing protein in a single intervention, instead of repeated dosing, which could mean a significant improvement in quality of life.
“This first ATMP classification for our platform technology is an important step in engaging with regulatory authorities for this entirely new category of genetically engineered cell factory therapeutics and is a critical step toward commercialization in Europe for advanced therapies like SIG-003,” Paul Wotton, PhD, chief executive officer of Sigilon, said in a press release. “As we proceed in development, it is critical that we work with regulators to define a clear path toward market approval from the earliest stages, which includes satisfying established international guidelines and standards for the future introduction of new Living Therapeutics.”
ATMP designations are granted for new therapies that fulfill the definitions for medicinal products that are gene therapy, somatic cell therapy, and/or tissue engineered products. The ATMP classification provides regulatory guidelines for preclinical development, manufacturing, and quality testing. The status also grants financial incentives such as fee reductions and other benefits.