Cell and Gene Therapy

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.

After years of quiet maturation, new approach methodologies and emerging clinical success for cell therapies are fueling parallel momentum for induced pluripotent stem cells.

A new biotech co-founded by Paul Stoffels has formed to take on the CAR T cell assets of Galapagos almost a year after the company—now known as Lakefront Biotherapeutics—abandoned them.

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.

Caribou Biosciences entered the scene in 2011 and went public in 2021. Over the course of its existence, the biotech has received funding and support from a handful of Big Pharma players, including Novartis, AbbVie and Pfizer.