LONDON – Bayer AG is making a major move into gene therapy with the $4 billion acquisition of one of the pioneers of the field, Asklepios Biopharmaceutical Inc. The deal will give the German pharma access to an adeno-associated viral vector platform that has generated multiple commercial and clinical stage assets across a broad range of indications from rare diseases to chronic conditions. The in-house portfolio includes treatments for Pompe and Parkinson’s diseases and congestive heart failure. Asklepios (Askbio) also has spun out programs in hemophilia and Duchenne muscular dystrophy. Read More
The fatty acid amide hydrolase (FAAH) inhibitor space may seem, in the words of one analyst, “historically a graveyard,” but that didn’t keep Jazz Pharmaceuticals plc from buying Springworks Therapeutics Inc.’s program with a plan to take aim first at post-traumatic stress disorder (PTSD) and associated symptoms. Read More
Intellia Therapeutics Inc. is looking to disrupt the transthyretin (TTR) amyloidosis (ATTR) market with NTLA-2001, its CRISPR-based treatment designed to be a potential cure for the disease. The drug, which is delivered via a lipid nanoparticle, edits the patient's DNA in vivo to create a stop codon and eliminate the expression of TTR, the protein that aggregates in ATTR patients' nervous systems and hearts, disrupting their functions. Read More
Mirati Therapeutics Inc.’s update on the phase I/II Krystal trial of the KRAS-G12C-targeting adagrasib (MRTX-849) was arguably the most eagerly awaited news, and certainly the most eagerly awaited KRAS-targeting news, to come out of the 2020 EORTC-NCI-AACR (ENA) Molecular Targets meeting. KRAS is one of the most frequently mutated oncogenes across a wide swath of solid tumors, and has been one of the toughest nuts to crack as far as druggability is concerned. Read More
Under the right circumstances, a single mouse can be as good as a group of eight or 10 animals in predicting whether a tumor will respond to a drug, researchers reported at the 2020 EORTC-NCI-AACR (ENA) Molecular Targets meeting on Saturday. The single-animal approach “allows incorporation of more tumor models within the same resource constraints,” Peter Houghton told reporters at a press conference previewing ENA highlights. Read More
Interest in testing the mettle of three widely available drugs against COVID-19 gained traction Monday, led by suggestions that daily low-dose aspirin might lower the risks of complications and death from the disease. Read More
The latest global regulatory news, changes and updates affecting biopharma, including: MOU to help with compounding oversight; MHRA advises on MA transition; CMS delays date for radiation oncology bundle; NRC issues advisory letter to INIS. Read More
New and updated preclinical and clinical data presented by biopharma firms at the 32nd EORTC-NCI-AACR Symposium, including: Aileron, Basilea, Black Diamond, Cyclacel, Elevation Oncology, Essa, Exelixis, Kura, Medicenna, Mirati, Rain, Revolution, Syros. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Allakos, Anaptysbio, Astrazeneca, Biovaxys, Cohbar, Collegium, Contrafect, Cytodyn, Geovax, GSK, Lidds, Moderna, Novartis, Olema, Quell, Resverlogix, Revolution, TFF, Titan. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aeglea, Bayer, Bioinvent, Bioxcel, Forte, Janssen, Nicox, Nitric Oxide Innovations, Passage, Santen, Sesen, Spectrum, Tracon, Y-mabs. Read More
