Cell and Gene Therapy
THE LATEST
In this episode of Denatured, you’ll hear from Jerome Barley, commercial director at Avion Medical and Ryan Klein, vice president, business development, US and APAC at Resolian. We discuss why Australia has become a magnet for early clinical development, and whether its speed, regulatory clarity and tax incentives are pushing it toward a more integrated biotech role.
The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.
Merck stole the show at the European Society for Medical Oncology (ESMO) annual conference, with new data on its Moderna-partnered personalized cancer vaccine leaking prior to the presentation and antibody-drug conjugate sac-TMT making a splash in endometrial cancer; Bristol Myers Squibb revealed the death of a patient with multiple sclerosis in an early-stage trial of zola-cel; and pharma is on track to easily surpass last year’s deal count.
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.
The disclosure—which came ahead of a presentation of data from the Phase 1 Breakfree-2 study at the ACTRIMS-ECTRIMS meeting—follows Bristol Myers Squibb’s late summer revelation that it had paused certain trials of zolacabtagene autoleucel.
Aside from RG6540, Roche also discontinued a host of other early-stage assets, including an antisense oligonucleotide for Alzheimer’s disease and a small-molecule candidate for geographic atrophy.
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.
As trispecifics, cell engagers and other complex formats multiply, drug developers are seeking discovery and engineering partners to help navigate an expanding universe of molecular possibilities.