CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sigilon Therapeutics announced today that it will present data at the American Society of Hematology Annual Meeting demonstrating that its novel Shielded Living TherapeuticsTM platform for rare bleeding disorders remains viable in animal models for at least six months. The data also demonstrate dose-responsive in vivo expression of human coagulation factor VIII (hFVIII) and correction of the bleeding phenotype in immunocompetent hemophilia A mice.
“The data we will be sharing at ASH are another important validation of our Shielded Living Therapeutics platform and our ability to deliver sustained production of crucial proteins, such as factor VIII, with a single treatment,” said David Moller, M.D., Sigilon’s Chief Scientific Officer.
The ASH abstract details Sigilon’s novel approach to developing durable cellular therapies. The process starts with engineering human cell lines to express high levels of hFVIII without the use of viral vectors. These cells are then encapsulated inside dual-compartment spheres. The inner compartment is designed to maximize cell viability and protein production in vivo. The outer layer of the spheres contains proprietary small molecules conjugated to alginate which avoids fibrosis and immune system attack.
In the latest studies, the therapeutic cells remained viable and continued producing stable levels of hFVIII six months after implantation in mice, after which the study was terminated. If these data are replicated in the clinic, the implanted cells could potentially eliminate the need for patients to get regular factor or non-factor injections.
“These data are unprecedented in demonstrating sustained expression of therapeutic proteins for the treatment of hemophilia,” said Deya Corzo, M.D., Sigilon’s Chief Medical Officer. “The results bolster our confidence in our Shielded Living Therapeutics platform, which enables a modular approach to harnessing the body’s most powerful machine – the human cell – to express therapeutic proteins for a wide array of indications.”
Sigilon recently received Orphan Drug Designation for SIG-001, its investigational therapy for hemophilia A. Clinical trials of SIG-001 are expected to begin in 2020. “We are looking forward to moving SIG-001 into the clinic as we advance our mission to replace fear with hope in patients living with chronic disease,” Dr. Corzo said.
The ASH abstract 2065 is entitled: “Correcting Rare Blood Disorders Using Coagulation Factors Produced in vivo by Shielded Living Therapeutics™ Products” and will be presented on Saturday, Dec. 7 from 5:30 – 7:30 p.m. ET during the Gene Therapy and Transfer: Poster I Session in Hall B of the Orange County Convention Center.
About Sigilon Therapeutics
Sigilon Therapeutics is developing functional cures for chronic diseases through its Shielded Living Therapeutics™ platform. Sigilon’s therapeutics consist of novel human cells engineered to produce the crucial proteins, enzymes or factors needed by patients living with chronic diseases such as hemophilia, diabetes and lysosomal storage disorders. The engineered cells are protected by Sigilon’s Afibromer™ biomaterials matrix, which shields them from immune rejection and fibrosis. Sigilon was founded by Flagship Pioneering in conjunction with Daniel Anderson, Ph.D., and Robert Langer, Sc.D., of the Massachusetts Institute of Technology.