SiSaf gets go-ahead from FDA to treat marble disease

The ODD from the FDA provides SiSaf with incentives such as tax credits for clinical trials, waivers of user fees, and increased market exclusivity.

In addition, the PRDD has granted SiSaf the right to apply for a Priority Review Voucher that can be used by the FDA to expedite the drug approval process.

Sisaf’s treatment treats the serious or life-threatening conditions of autosomal dominant osteopetrosis type 2 (ADO2), which primarily affects children, including blindness due to optic nerve compression, abnormal development of teeth and craniofacial scoliosis, and scoliosis. was sentenced to PRDD on grounds.

There is currently no approved treatment for granulosis ADO2, nor are there other treatments currently in clinical trials.

Therefore, if approved, SiSaf’s SIS-101-ADO could be the first treatment for granuloma ADO2 and bring life-changing benefits to those suffering from this debilitating disease.

SiSaf is currently preparing for first-in-human clinical trials.


SIS-101-ADO combines siRNA that suppresses CLCN7 expression with SiSaf’s Bio-Courier delivery technology.

RNA therapy restores bone mass and quality to near-normal levels by downregulating the expression of CLCN7, a mutated gene expressed by osteoclasts and other cell types responsible for ADO2. .

Inherited skeletal disorders such as ADO2 account for 5% of all birth defects worldwide, yet many unmet needs and challenges remain in providing safe and effective treatments. I’m here.

SiSaf says its proprietary Bio-Courier technology could accelerate the development of new RNA-based therapeutics. This technology addresses the limitations of other RNA delivery techniques by stabilizing lipid nanoparticles with bioabsorbable silicon.

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