SiSaf reveals positive preclinical data in siRNA program to treat osteopetrosis


SiSaf Ltd, an RNA delivery and therapeutics company, has announced positive data confirming safety and efficacy of its Bio-Courier next generation silicon stabilized hybrid lipid nanoparticles (sshLNP) as an effective non-viral delivery system for autosomal dominant osteopetrosis type 2 (ADO2) siRNA therapy.

The data was presented at the American Society of Bone and Mineral Research (ASBMR) 2022 annual meeting in Austin, Texas, by SiSaf’s collaborators from the University of L’Aquila, Italy.

SiSaf’s lead in house program, SIS-101-ADO, is an ADO2-specific siRNA combined with SiSaf’s Bio-Courier platform, which aims to suppress the expression of the mutant CLCN7 gene and thereby rescue bone mass and quality to nearly normal levels. 

The new data confirmed that sshLNPs can be designed for specific cell lines using different formulations. It also confirmed sshLNP is an effective non-viral delivery system for AD02, with the data showing down regulation of the CLCN7 gene. Also, the data confirms the safety profile of siRNA delivered by the sshLNP technology in an animal model, with no side effects at the end of the full-length study.

ADO2 (also known as OPTA2 or Albers-Schonberg disease)

Osteopetrosis refers to a group of rare, inherited skeletal disorders characterized by increased bone density and abnormal bone growth.

Symptoms and severity can vary, ranging from neonatal onset with life-threatening complications such as bone marrow failure. Depending on severity and age, those with the condition may have a variety of issues including fractures, short stature, compressive neuropathies (pressure on the nerves).

Suzanne Saffie-Siebert, CEO of SiSaf Ltd said: “Skeletal rare diseases still pose unmet therapeutic needs as the major challenge related to intrinsic difficulty in targeting bone. SiSaf’s silicon stabilized hybrid lipid nanoparticles constitute a next-generation non-viral vector able to retain both integrity and stability of constructs over time and offer considerable payloads of biologicals without requiring any cold-chain distribution.”

Antonio Maurizi from Anna Maria Teti’s team in the Department of Biotechnological and Applied Clinical Sciences said: “Given obtained results, SiSaf sshLNP constitutes the most promising bone targeting non-viral vector available for addressing rare skeletal disorders by safely and effectively delivering Biologicals of interest.”

SiSaf study preparation

SiSaf is developing the trial design and preparing the IND data package for a planned phase I clinical study of SIS-101-ADO, with the aim of submitting an application to the U.S. FDA within the next six months.

SiSaf has an ongoing collaboration with Teti’s group and is working to extend its current pipeline of Bio-Courier targeted siRNA therapies to cure other genetic skeletal disorders. Its strategy is to develop its pipeline of novel therapies through clinical studies and then seek collaborative partnerships with marketing expertise in genetic skeletal disorders and rare diseases.

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