The trial is expected to begin by June. Last August, the U.S. Food and Drug Administration (FDA) granted the potential cell therapy orphan drug status, a designation that promotes rare disease treatment development through benefits including a tax credit for trial expenses, waived user fees, and marketing exclusivity.
The company said it will also use the funds to advance and expand its programs in rare blood disorders and other diseases. The programs are based on the biotechnology company’s proprietary Shielded Living Therapeutics platform.
New investors including Canada Pension Plan Investment Board, Longevity Vision Fund, and funds managed by BlackRock joined founding investor Flagship Pioneering and other investors, including Eli Lilly and Company, in the Series B financing round. To date, total Sigilon funding exceeds $195 million.
“Sigilon is driven to liberate patients from the fear of living with serious chronic diseases, and from alternative therapeutic approaches,” Rogerio Vivaldi, MD, president and chief executive officer at Sigilon, said in a press release.
“Our Shielded Living Therapeutics platform is designed to give patients who have chronic diseases a convenient, safe, long-term therapeutic benefit,” Vivaldi said. “We believe encapsulating engineered human cells in our proprietary matrix will enable us to deliver controlled doses of therapeutic proteins without the need for immunosuppression and without the risks associated with modifying patients’ genomes.”
SIG-001 is made up of human cells engineered to incorporate artificial DNA-encoding therapeutic proteins into cells, specifically human FVIII, which is missing in people with hemophilia A.
The modified cells are protected from immune rejection and tissue scarring (fibrosis) by Sigilon’s Afibromer artificial biomaterials matrix. In addition, the biomaterial improves the optimized delivery and long-term stability of the cells in the body. After a single implant, it is expected the modified cells can foster long-term creation of therapeutic proteins.
In preclinical studies, delivery of SIG-001 into a mouse model of hemophilia A resulted in a steady production of FVIII, and controlled bleeding. This was also the case with animal models of hemophilia B and factor VII deficiency.
The studies demonstrated that delivery of different doses of the therapy to an animal’s abdomen led to a dose-dependent production of FVIII. SIG-001 also helped curb bleeding and blood loss following a bleeding test, and kept FVIII levels stable for more than six months.
“Sigilon’s Shielded Living Therapeutics platform offers patients with chronic disease the prospect of relief without disrupting their lives,” said Douglas Cole, MD, Sigilon chairman. “The near-term transition to clinical development and the platform’s breadth and progress reflect the power and productivity of Sigilon’s approach.”
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