To get an insight into what regulatory agencies are looking for demonstrating safety for anticancer CGTs, we analyzed the supporting nonclinical studies for the currently approved therapies.Methods:We reviewed of the European Public Assessment Reports (EPAR) and FDA Summary Basis for Approval (SBA) ...
It offers an efficient approach to develop genetically modified animal models and a potential strategy for targeted gene therapies. The CRISPR/Cas9 system allows simultaneous digestion of multiple targets at multiple sites in the same cell, making it possible to knock out or knock in multiple genes...
The FDA approval of the enzyme replacement therapy cerliponase alfa (Brineura) for the treatment of CLN2 Batten disease is an important milestone in Batten disease therapy. Promising results from preclinical research indicate that gene therapy — particularly approaches that use adeno-associated virus ...
We shall also briefly reflect upon the recent advancements of the exosome-liposome hybrids in cancer therapy. Finally, we put forward future directions for the use of exosome/liposome and/or hybrid nanocarriers for accurate diagnosis and personalized therapies for cancers....
Bioengineering has resulted in the development of multiple devices to treat various neurological diseases with many now approved by the FDA [124]. There are two promising devices under development to treat PMD: near-infrared light-emitting diode (NIR-LED) and transcranial direct current stimulation ...
can be used as gain-of-function therapies, replacing non-functional mutant proteins to restore normal physiological function.BThe table shows U.S. FDA-approved RNAi drugs currently in clinical use Full size image Vaccines for infectious diseases...
(d-Ala–d-Ala ligase) alleles and the presence of a CRISPR-Cas system, while in the second strain, it was the presence of the genepbp5which can confer ampicillin resistance. Clade-specific traits exist; clade B isolates encode several secreted factors that can interact with eukaryotic cell ...
• Exploration to other indication can only be approved if the clinical MOA is similar.• PMS is mandatory.• Immunogenicity is mandatory. [EMEA – European Medicines Agency; GMP – Good Manufacturing Practices; ICH – International Committee on Harmonization; DCGI – Drug Controller General of...
The clinical application of CAR-T cell therapy has yielded remarkable success in treating certain hematological malignancies, such as relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), diffuse large B-cell lymphoma (DLBCL) and multiple myeloma [9,10]. FDA-approved therapies like tisa...
Infliximab approved for use in Crohn’s disease: a report on the FDA GI Advisory Committee conference. Inflamm. Bowel Dis. 4, 328–329 (1998). Article CAS PubMed Google Scholar Morrow, T. & Felcone, L. H. Defining the difference: what makes biologics unique. Biotechnol. Healthc. 1,...