Ark's gene therapy stumbles at the finish line
- MedicineNature Biotechnology
The company still hopes the EMEA decision to reject Cerepro may be reversed on appeal, which is in process and likely to be considered next month, and has several other gene therapy products well advanced in its pipeline—some of them, according to financial analysts, even more promising than Cerepro, which had anticipated worldwide annual sales of $200 million.
Gene therapy and virotherapy: novel therapeutic approaches for brain tumors.
- Biology, MedicineDiscovery medicine
This review will discuss various virotherapy and gene therapy approaches for GBM currently under pre-clinical and clinical evaluation including direct or conditional cytotoxic, and/or immunostimulatory approaches.
Development of Gene Therapeutics for Head and Neck Cancer in China: From Bench to Bedside.
- Medicine, Biology
Two gene therapy drugs, Gendicine and Oncorine, have been marketed in China, and a number of upcoming gene therapy agents are under development for the treatment of head and neck cancer.
Viral vector-mediated gene transfer for CNS disease
- BiologyExpert opinion on biological therapy
Although the majority of clinical trials have centered on gene replacement and neuroprotection approaches, the field is advancing in the direction of neuromodulation, gene silencing and other newer strategies.
Synthetic mRNA nanoparticle-mediated restoration of p53 tumor suppressor sensitizes p53-deficient cancers to mTOR inhibition
- Biology, ChemistryScience Translational Medicine
Redox-sensitive mRNA nanoparticles help kill tumors by restoring p53 expression by delivering p53-mRNA in nanoparticles and indicate that restoration of tumor suppressors by the synthetic mRNA NP delivery strategy could be combined together with other therapies for potent combinatorial cancer treatment.
Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework
- MedicineFront. Med.
The current European legal framework of ATMPs is reviewed, an overview of the clinical applications for approved and investigational GT MPs is provided, and critical challenges in the development of GTMPs are discussed.
SHOWING 1-2 OF 2 REFERENCES
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
- Biology, MedicineNature Medicine
We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic…
Gene therapy: cursed or inching towards credibility?
- ArtNature Biotechnology
A chastened but determined group of pioneers believes gene therapy can be lived down and live up to its initial promise, and they are pointing to a new generation of products to back up that claim.