High cost is a major challenge. The global cell and gene therapy market is projected to expand by $6.56b, reflecting an ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...

(Yicai) Nov. 15 -- The Germany’ Merck and five other overseas pharmaceutical companies have been given the green light to ...

The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...

At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...

The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency. It ...

Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...

Oncology Focus : CAR-T and CAR-NK therapies represent ~30% of all cell and gene trials, with a strong focus on solid and hematological malignancies. Significant attention on gastrointestinal tract and ...

The city's first guide to quality management of cell therapy drugs used in medical institutions was released on Tuesday, ...