Gene therapy
As both Taysha Gene Therapies and Neurogene warm up for an important year of readouts, the Rett syndrome community is hopeful of seeing a therapy that can truly change the disease’s path.
CROs cite faster startup timelines, regulatory predictability and tax incentives as reasons for growing interest in Australia. State investments in manufacturing, translational research and advanced therapies aim to expand that advantage into a broader biotech ecosystem. The question remains whether Australia can support drug development at broader scale.
In this episode of Denatured, you’ll hear from Bruce Culleton, CEO at ProKidney and Demetri Maxim, CEO at Nephrogen. We discuss why kidney disease has long been a hard target for cell and gene therapies, and what new delivery strategies, therapeutic approaches and regulatory clarity mean for commercially viable products.
METiS TechBio’s AiLNP platform uses machine learning models to develop lipid nanoparticles with potentially better tissue specificity that could enhance a drug’s therapeutic performance.
CDMO-sponsor relationships have shifted from transactional outsourcing to strategic collaboration built on shared risk, regulatory fluency and technical insight. As rising compliance pressure and geopolitical volatility reshape manufacturing, partners like Rose BioSolutions argue that true value now comes from challenging assumptions, anticipating hurdles and protecting programs before problems surface.
Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.
A surge of consolidation, rising appetite for specialized manufacturing and a shift toward decentralized trials is reshaping Switzerland’s life sciences landscape. From peptide producers to organoid CROs, service providers are scaling and diversifying as global sponsors turn to Swiss hubs for talent, innovation and regulatory strength.
Jefferies analysts made the prediction after Sarepta Therapeutics, which has endured nothing short of a saga with the Duchenne muscular dystrophy gene therapy Elevidys, presented data this week showing its clinically meaningful benefit in older patients who are able to walk.
The market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene therapy last month.
Blood centers have the donors, resources and community footprint drug sponsors need to solve supply chain issues around cell and gene therapies.
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