Purespring Therapeutics Ltd. has raised £80 million (US$104.6 million) in a series B, putting it on course to be the first to take a gene therapy for a kidney disease into the clinic. The money enables the company to move the lead program, PS-002, for the treatment of IgA nephropathy to clinical proof of concept and advance programs in other complement-mediated kidney diseases, and in an undisclosed glomerular kidney disease.

Zealand gets a second CRL in ultra-rare disease

U.S. FDA has issued yet another complete response letter (CRL) for dasiglucagon, a glucagon receptor agonist, being developed by Zealand Pharma A/S for treating congenital hyperinsulinism. This CRL is pegged to the timing of a third-party manufacturing facility reinspection that was done in August and September. A new inspection classification is pending, the company said. In December, the FDA issued the company a CRL for deficiencies it found following an inspection at a third-party contract manufacturing facility. Severe and ultra rare disease is generally defined as affecting fewer than one in every 50,000 people. Two other companies, Rezolute Inc. and Amylyx Pharmaceuticals Inc., are developing congenital hyperinsulinism therapies.

Chemistry Nobel awarded for 3D protein design, prediction work

David Baker, Demis Hassabis and John Jumper share the 2024 Nobel Prize in Chemistry for their contributions to the science of protein structure. David Baker was awarded half the prize “for computational protein design,” according to the Royal Swedish Academy of Sciences. Hassabis and Jumper shared the other half “for protein structure prediction.” After being named a 2024 Citation Laureate by Clarivate Analytics in September, Baker sat down with BioWorld to talk about his work, explaining these two concepts.

Complete Genomics seeks way out of Biosecure Act

One of the companies explicitly named in the Biosecure Act, which has yet to be passed by the U.S. Senate, is fighting back. Raising the possibility of a constitutional challenge to the bill, Rade Drmanac, co-founder and chief scientific officer of Complete Genomics, wrote to congressional leaders to urge them to remove the California-based subsidiary of MGI from the bill. “We share the desire of many in Congress to protect personal DNA data and diversify the American supply chain, but singling out companies like ours that don’t even have access to such data – and therefore cannot share it . . . is not the way to accomplish this,” he said. A manufacturer of genomic sequencing instruments, Complete Genomics was included in the bill based on false information and given no chance to dispute it, Drmanac said.

Japan pledges to become ‘land of drug discovery’: Bio Japan 2024 

The Japanese government is throwing full weight behind the Bioeconomy Strategy initiative to position Japan’s biopharmaceutical industry as both a key driver of economic growth and global drug discovery hub. “2024 and 2025 [are] milestone years to deepen cooperation and accelerate efforts of Japan’s Bioeconomy Strategy as well as [the country’s reformed] health care policy,” Kito Masahiro, of Japan Pharmaceutical Manufacturers Association (JPMA), said at Bio Japan 2024, which kicked off at the Pacifico Yokohama convention center Oct. 9. Kito noted that 2024 marks a point of reflection for policy makers, industry and academia as Japan turns a corner in the Bioeconomy Strategy, first introduced in 2019, as it heads toward the 2030 goal of making Japan ”the world’s most advanced bioeconomy.”

SK Bioscience to invest $3M in US vaccine maker Fina Biosolutions 

SK Bioscience Co. Ltd., of Seongnam-si, South Korea, said Oct. 8 that it will acquire a $3 million stake in Fina Biosolutions LLC to become the first and sole strategic investor of the Rockville, Md.-based vaccine specialist. For SK Bioscience, the investment is slated to boost its vaccine R&D capabilities, the company said, which currently includes manufacturing of pneumococcal and typhoid conjugate vaccines. “Through our mid- to long-term collaboration with Fina, we will advance the vaccines we are developing to the next level and strengthen our competitiveness for global market expansion,” SK Bioscience CEO and President Jaeyong Ahn said.

BioWorld Insider podcast: Gene and cell therapies will propel innovation, says Astellas CCO 

Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in this episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.” Zieler also shared his thoughts on creating sustainable health care in aging societies, the innovation cycle gap between the U.S. and Europe, and how collaboration between business and government is critical in forging ahead in this wide-ranging discussion.

Also in the news

Ask, Autonomous, Bayer, Biogen, Black Diamond, Blue Earth, Calluna, City, Curve, Eisai, Fina, Gilead, GSK, Innovent, Kiromic, Lipocine, Merck, Mink, Moma, Neuvivo, Orasis, Pharmalink, Pharmanovia, PTC, Relief, Sage, Satsuma, Scholar Rock, Scilex, Siga, Soleno, Somagenetix, Teva, Tonix, Vincerx, X-Chem, Zealand