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
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.
“The granting of Orphan Drug Designation and Rare Pediatric Disease Designation is a major milestone in our efforts to advance innovative siRNA therapeutics to alleviate the pain and suffering caused by granuloma ADO2,” said SiSaf Founder. Dr. Suzanne Saffie Sievert, CEO and CEO, said. .
“SIS-101-ADO opens the door to a new era of personalized care and treatment options for ADO2 and other rare bone and skeletal diseases.
“Interest in RNA therapeutics for a wide range of medical conditions is exploding, and siRNA technology from SiSaf’s innovative biocourier has the potential to address some of the most intractable diseases. ”
SIS-101-ADO and other BioCourier combinations could not only treat rare skeletal disorders, but could also pave the way for other rare diseases previously thought to be untreatable. Yes, added Safi Siebert.