Pelacarsen

Today, a brief rundown of news from The Royal Swedish Academy of Sciences and Alnylam Pharmaceuticals, as well as updates from Astellas, Johnson & Johnson and AstraZeneca that you may have missed.David Baker, Demis Hassabis and John Jumper won this years Nobel Prize in chemistry for their respective work on computational protein design and protein structure prediction. A serial entrepreneur, Baker spent decades studying proteins, knowledge that helped him and his team to design unique protein structures. Hassabis and Jumper, both employees of Googles DeepMind group, developed an artificial intelligence model, dubbed AlphaFold, that could be used to predict the structure of virtually all the 200 million or so proteins known to science. Together, their work has helped solve the protein folding problem, which could allow researchers to design better drugs. Ned PagliaruloAlnylam Pharmaceuticals has submitted its medicine vutrisiran for a second approval from the Food and Drug Administration. Vutrisiran is already available in the U.S., sold under the brand name Amvuttra, as a treatment for nerve damage associated with the inherited form of a rare disease called transthyretin amyloidosis. Alnylam is now trying to get the medicine cleared for use in another form of the disease hallmarked by serious, potentially fatal heart problems. The company is using a regulatory fast pass to speed the FDAs review. If approved, vutrisiran would directly compete against a blockbuster Pfizer drug and, potentially, an under-review therapy from BridgeBio Pharma. Jacob BellJohnson & Johnson said Monday it is terminating a trial of its drug delivery system TAR-200 in bladder cancer because treatment didnt outperform a combination of chemotherapy and radiation. The Sunrise-2 trial tested TAR-200, a small device that secretes the chemotherapy gemcitabine, and chemoradiation in people whose bladder cancer had spread into surrounding muscle tissue and who arent having their cancerous tissue removed with surgery. J&J has multiple other studies of TAR-200 underway and plans to ask the Food and Drug Administration for approval next year based on the results of one completed study. Jonathan GardnerGemma Biotherapeutics, a biotechnology company founded by gene therapy pioneer Jim Wilson, is partnering with a public health research institution in Brazils Ministry of Health. Gemma says the institution, known as Fiocruz, has extensive experience manufacturing drugs, and is the main supplier of immunobiologics, biopharmaceuticals and diagnostics to the ministry. The agreement has Fiocruz providing up to $100 million, which will be used for clinical research and manufacturing, with the ultimate goal of more widely distributing rare disease gene therapies in Brazil at a more affordable cost. Jacob BellAstraZeneca said Monday it is paying Hong Kong-based drugmaker CPSC Pharmaceutical Group $100 million for a preclinical oral cholesterol-lowering drug. The drug, codenamed YS2302018, blocks a lipoprotein called Lp(a) that is implicated in cardiovascular disease and is slightly raised by statins. Lp(a) is targeted by other drugs in development such as Amgens olpasiran, Eli Lillys lepodisiran and Ionis and Novartis pelacarsen. Per deal terms, CPSC is eligible for up to $1.9 billion in additional payments based on achievement of development and commercial milestones. Jonathan GardnerJapan-based drugmaker Astellas has bought rights to an experimental gene therapy through a heavily backloaded deal announced Tuesday. Upfront, Astellas is investing $20 million in the therapys developer, AviadoBio, and providing a payment of up to $30 million. But if the program hits certain goals, AviadoBio could take home up to $2.18 billion more through milestone payments and additional licensing fees. The therapy is currently being evaluated in a small clinical trial, as a potential treatment for certain patients with frontotemporal dementia. Jacob Bell '