As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene ...

Battling a rare mutation of cystic fibrosis, Emily Kramer-Golinkoff fights for her life — and for gene therapies that offer ...

People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

"Moreover, the duration of follow-up is among the longest reported for a lentiviral vector-based gene therapy; 13 patients ...

Crispr Therapeutics is an emerging gene editing company focused on the development of Crispr/Cas9-based therapeutics. The company's proprietary platform specializes in clustered regularly interspaced ...

Biotech firms are engineering smarter, safer gene delivery systems to enhance the safety and efficacy of genetic medicines.

Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 ...

Benjamin K. Chen, MD, PhD, discussed the next steps after the results of his study in genetic tagging showed promise in ...

South Korea-based DAAN Biotherapeutics has entered a licensing agreement with GC Cell, granting the latter exclusive rights ...

A new way to deliver disease-fighting proteins throughout the brain may improve the treatment of Alzheimer's disease and other neurological disorders, according to scientists. By engineering human ...

So says, Gerhard Bauer, ex-director of the UC Davis GMP manufacturing facility and co-author of a new study examining cell and gene therapy production in the United States. “New manufacturing ...