Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
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.
There was more juice to be extracted from the grape that was the very successful Chinook Therapeutics Inc., so two investors have decided to make the most of it. Novartis AG and Versant Ventures have launched Borealis Biosciences Inc. from Chinook’s remnants to develop RNA therapies for treating kidney disease.
In a deal that could bring Avidity Biosciences Inc. $2.3 billion if all milestones are met, Bristol Myers Squibb Co. gained global rights to Avidity’s antibody oligonucleotide conjugates platform to advance up to five genetic cardiovascular targets. Avidity’s technology, which combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, aims to address the root cause of diseases that are untreatable with current RNA therapeutics. Its lead internal programs are based on the targeted delivery of RNA into muscle.
In its second big pharma deal to date, Shape Therapeutics Inc. drew Otsuka Pharmaceutical Co. Ltd. to the table in a potential $1.5 billion-plus collaboration initially aimed at developing gene therapies for ocular diseases. The multitarget agreement, which includes options for additional targets and tissue types, will combine Shape’s AI-driven adeno-associated virus (AAV) platform and Otsuka’s expertise in ophthalmology to develop intravitreally delivered AAV therapies.
Launching with a $300 million series A financing, Renagade Therapeutics Inc. has set out to target disease on a large scale – at every point throughout the human body where disease forms – through its RNA platform designed to deliver, code, edit and insert genetic information. The round, led by founding investors MPM Bioimpact and F2 Ventures, is the largest venture capital (VC) round for a U.S.-based biopharma company this year, and it is among the top series A rounds for the industry to date.
Orbital Therapeutics Inc. raised $270 million in a series A round to fund a big push into the next generation of mRNA-based therapies. The Cambridge, Mass.-based firm is building out a comprehensive RNA platform from which it will launch programs in oncology, autoimmune disease and indications involving protein replacement approaches.
Acute thrombosis, including heart attack and stroke, is a leading cause of mortality worldwide. Yet only a small fraction of patients can be treated with current therapeutic or surgical interventions. Enter Basking Biosciences Inc., a 2019 startup aimed at developing a short-acting, fast-onset thrombolytic drug alongside a reversal agent for treating acute ischemic stroke.
Shares of Avidity Biosciences Inc. (NASDAQ:RNA) dropped 15% March 30 as the company disclosed details of the serious adverse event (SAE) that prompted a partial clinical hold in September 2023 on the phase I/II trial testing AOC-1001 in adults with myotonic dystrophy type 1 (DM1).
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
