Cell and Gene Therapy
THE LATEST
Stem cell therapies vary widely yet many physicians still treat them as interchangeable. A more credible model requires diagnostics, product characterization and conventional medical context to determine who and when should receive regenerative treatment.
Ten patients out of 20 who received Johnson & Johnson and Legend Biotech’s Carvykti remained alive at five years of follow up.
Kyverna Therapeutics’ miv-cel showed improved mobility and a favorable safety profile at one year in patients with stiff person syndrome, further derisking an upcoming FDA application, William Blair said on Thursday. The biotech plans to file for priority review this year.
Novo plans to hit $23B in revenue by 2035, has further reduced its workforce by 4,000 and is boosting manufacturing capacity for its Wegovy pill, but investors are unconvinced it will be enough to turn things around; Viking Therapeutics and Roche report positive results for their weight loss assets; Ultragenyx snags FDA approval for its Sanfilippo syndrome gene therapy; IPOs keep on coming.
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.
The FDA held a public forum on Monday to discuss the future of psychedelic therapeutics, followed by a meeting on Wednesday regarding recommendations for reauthorizing the Prescription Drug User Fee Act the day after launching its TrialBlazer initiative. Meanwhile, Definium Therapeutics notched another late-stage trial win for its LSD drug, and Novartis continues to reel from the company’s triple asset stumble.
Blood centers have the donors, resources and community footprint drug sponsors need to solve supply chain issues around cell and gene therapies.
Encoded Therapeutics’ lead asset ETX101 is a gene therapy being tested for Dravet syndrome. Phase 1/2 data showed substantial reductions in seizures.
In this episode of Denatured, sponsored by SK pharmteco, Jennifer Smith-Parker speaks to Arvind Singh, vice president and global head of supply chain. In this episode, we explore how CDMOs are reshaping supply‑chain strategy for a new generation of complex modalities.
September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.