After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
At Bio Japan 2024, policymakers and industry leaders pledged to make Japan a land of drug discovery to attract native and foreign drug developers. Like many Asian countries, Japan is harnessing and prioritizing the bio sector to drive economic growth, throwing its weight behind its 10-year Bioeconomy initiative to create bioclusters and increase investment.
Sangamo Therapeutics Inc.’s second large, worldwide licensing deal for its capsid technology in the past five months is with Astellas Pharma Inc. The California-based company is getting $20 million up front and the chance to bring in up to $1.3 billion in fees and milestone payments in an agreement spanning five potential disease targets for gene therapies to treat neurological diseases.
Gene therapy faces complexities in delivering treatments due to persistent safety concerns and daunting immune responses, but Next Generation Gene Therapeutics Inc. has found a way around this issue using dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function.
Ring Therapeutics Inc. has joined forces with Singapore’s Agency for Science, Technology, and Research and with the Singapore Eye Research Institute to advance the first new class of viral vectors in more than 50 years, Ring CEO Tuyen Ong told BioWorld.
Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) set up its second overseas regulatory office in Washington, four months after the drug and med-tech regulator opened its first Asia base in Bangkok, Thailand, in July 2024.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.
Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in the newest 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.”
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