Cell and Gene Therapy
THE LATEST
Sana Biotechnology is looking to start clinical development for its type 1 diabetes therapy SC451 this year.
The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.
Dozens of biotechs reported earnings this week. BioSpace recaps key highlights from Capricor Therapeutics, Legend Biotech, Inovio and Allogene.
Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question whether shared evidence and prior knowledge will accelerate development in rare diseases.
Analysts expect the market for manufacturing cell and gene therapies, worth less than $20 billion in 2024, to expand rapidly as approvals drive higher volumes of production.
Stylus Medicine, a member of BioSpace’s NextGen Class of 2026, launched in May 2025 to develop new, less complex genetic medicines. The company’s in vivo approach has attracted “intense” interest from Big Pharma.
UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp.
UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.
The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.