Avidity Biosciences Inc. has announced two new precision cardiology development candidates targeting rare genetic cardiomyopathies. AOC-1086 targets phospholamban (PLN) cardiomyopathy and AOC-1072 targets protein kinase AMP-activated noncatalytic subunit γ2 (PRKAG2) syndrome.
While RNA-medicine developer Wave Life Sciences Ltd. brought in a clinical data win, it also got knocked back a step as a major collaborator will go its separate way. That didn’t stop Wave’s stock from standing strong on the day. The company’s ongoing phase Ib/IIa study of its A-to-I RNA editing oligonucleotide produced positive proof-of-mechanism data in treating alpha-1 antitrypsin deficiency, a rare, genetic condition that can lead to lung and liver disease.
The founding CEO of Alnylam Pharmaceuticals Inc. is now leading the charge with newly launched City Therapeutics Inc., which just completed a $135 million series A financing. City’s executive chair, John Maraganore, will be in familiar territory as the new company plans to develop RNAi-based medicines using next-generation siRNA engineering. He expects dozens of these therapies to reach the market in a relatively short period of time, not just from City Therapeutics but from other companies. It’s a period in the development timeline that he finds reminiscent of the rise and development of monoclonal antibodies.
The founding CEO of Alnylam Pharmaceuticals Inc. is now leading the charge with newly launched City Therapeutics Inc., which just completed a $135 million series A financing. City’s executive chair, John Maraganore, will be in familiar territory as the new company plans to develop RNAi-based medicines using next-generation siRNA engineering. He expects dozens of these therapies to reach the market in a relatively short period of time, not just from City Therapeutics but from other companies. It’s a period in the development timeline that he finds reminiscent of the rise and development of monoclonal antibodies.
Judo Bio Inc. emerged from stealth mode and rolled out data showing the value of using megalin receptors for intracellular delivery of ligand-small interfering RNA (siRNA) therapeutics to the kidney as a way of reducing expression of the targeted genes.
Arrowhead Pharmaceuticals Inc. has filed for regulatory clearance to initiate a phase I/IIa trial of ARO-INHBE, the company’s investigational RNA interference (RNAi) therapeutic being developed for obesity.
Researchers from Imperial College London presented data from a study that aimed to assess the role of miR-1271-5p in prostate cancer (PCa) progression.
Researchers from Janssen Research & Development LLC presented preclinical data for JNJ-75220795 (ARO-PNPLA3), a GalNAc-conjugated small interfering RNA (siRNA) therapeutic targeting the PNPLA3 gene, being developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
The dark matter of long non-coding RNA (lncRNA) is shades brighter, after the signing of two major deals between biotech pioneers and big pharma in the past week. Haya Therapeutics SA announced Sept. 4 that it has sealed a multiyear agreement with Eli Lilly and Co. to apply its lncRNA platform technology to identify targets in obesity and related metabolic disorders.
The dark matter of long non-coding RNA (lncRNA) is shades brighter, after the signing of two major deals between biotech pioneers and big pharma in the past week. Haya Therapeutics SA announced Sept. 4 that it has sealed a multiyear agreement with Eli Lilly and Co. to apply its lncRNA platform technology to identify targets in obesity and related metabolic disorders.
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