One of the most important transformations in the pharmaceutical industry over the next decade is the ability to modify genes or expression and go after any target, Cargene Biopharma Inc. CEO Kathy He told BioWorld, explaining that small molecules and large molecules can only go after 15% of the known targets. But the technology platform of short-interfering RNA (siRNA) opens a huge opportunity to make those inaccessible targets available, she said.
One of the most important transformations in the pharmaceutical industry over the next decade is the ability to modify genes or expression and go after any target, Cargene Biopharma Inc. CEO Kathy He told BioWorld, explaining that small molecules and large molecules can only go after 15% of the known targets. But the technology platform of short-interfering RNA (siRNA) opens a huge opportunity to make those inaccessible targets available, she said.
Sirnaomics Ltd. is gearing up to start either an expanded phase IIb or a phase III clinical trial for its lead siRNA candidate, STP-705, for the treatment of two non-melanoma skin cancers: squamous cell carcinoma in situ and basal cell carcinoma.
Sirnaomics Ltd. is gearing up to start either an expanded phase IIb or a phase III clinical trial for its lead siRNA candidate, STP-705, for the treatment of two non-melanoma skin cancers: squamous cell carcinoma in situ and basal cell carcinoma. STP-705 comprises two siRNA oligonucleotides that inhibit transforming growth factor beta 1 and cyclooxygenase-2 gene expressions and is delivered via injection using what the company describes as a polypeptide nanoparticle-enhanced delivery mechanism.
Dysregulation of complement activation plays a key role in the pathophysiology of several diseases, which makes it an attractive therapeutic target. C3 has an important role and central position within the signaling cascade, and is a potential target for therapy.
Sirnaomics Ltd. went five for five in its study of treating cutaneous basal cell carcinoma. Five of five patients in a cohort of the open-label, dose-escalation phase II study of STP-705, composed of two sIRNA oligonucleotides targeting transforming growth factor beta 1 and cyclooxygenase-2, had a 100% complete response.
LONDON – The global pandemic has put the term mRNA on lips worldwide, but vaccines apart, the field of RNA therapeutics has failed to overcome the problem of delivery and is stuck at first base, targeting the liver. Newco Sixfold Bioscience Ltd. is the latest company working to end this constraint, applying a mixture of computation and advanced chemistry to design tissue-specific RNA delivery systems.
DUBLIN – Sisaf Ltd. has exercised an option on an Italian preclinical program to treat a rare bone disorder, autosomal dominant osteopetrosis type 2, with an siRNA molecule, which it will deliver with its in-house Bio-courier technology.
DUBLIN – Silence Therapeutics plc has entered the Chinese market through a partnering deal with Hansoh Pharmaceutical Group Co. Ltd. that involves developing short interfering RNA (siRNA) drugs against three undisclosed targets. The London-based firm is getting $16 million up front and could earn as much as $1.3 billion in development, regulatory and commercial milestones across the alliance. It will also receive tiered sales royalties, ranging from low double digits to mid-teens in percentage terms.
