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.
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.
DUBLIN – Ochre Bio Ltd. has raised $9.6 million in seed financing to drug the liver like never before, by combining genetics, single-cell genomics, spatial sequencing, imaging and machine learning to build a high-resolution, in silico model of the organ, which can provide new insights into disease biology and open up new drug targets.
DUBLIN – Auris Medical Holding Ltd., an early pioneer of targeted therapy for hearing disorders, is planning to exit the field and pivot into RNA-based therapeutics by acquiring a U.S. firm, Trasir Therapeutics Inc., which has developed an oligonucleotide delivery platform for delivery of short interfering RNA and other therapeutic RNA payloads to sites outside the liver.
HONG KONG – Olix Pharmaceuticals Inc. has entered an extensive research collaboration to test its RNAi therapeutic, OLX-104C, with Fimanac, PCI Biotech Holding ASA’s delivery technology for nucleic acid-based therapies, with the aim of reducing off-target effects while treating androgenic alopecia, or hair loss.
DUBLIN – Alnylam Pharmaceuticals Inc. is on track to secure its third FDA approval in successive years, as its siRNA drug, lumasiran, hit all its marks in a phase III trial in patients with primary hyperoxaluria type 1 (PH1). The drug is already undergoing regulatory review and has a Dec. 3 PDUFA action date. It is undergoing accelerated assessment in Europe as well.
DUBLIN – Silence Therapeutics plc entered a wide-ranging siRNA-based drug discovery and development deal with Astrazeneca plc that has propelled it toward the front ranks of siRNA platform companies. It is banking an up-front payment of $60 million, another $20 million as an equity investment and up to $400 million in option fees and milestone payments per candidate drug, in an alliance that could entail up to five active programs within three years.
