Therapies Related Topics 
Drug Discovery World
DECEMBER 19, 2023
The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.
Drug Discovery World
MAY 2, 2024
Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. Perhaps most significantly, Casgevy (or exa-cel) from Vertex Pharmaceuticals and CRISPR Therapeutics, was approved to treat sickle cell disease and became the first gene therapy that uses CRISPR gene editing.
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
Drug Discovery World
FEBRUARY 21, 2024
DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. PS : Most, if not all, current therapies are focused on MCI. RA: How does SNK01 work?
Drug Discovery World
JANUARY 5, 2024
CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body. “This very important study will answer whether CAR T-cell therapy offers a new treatment option for patients living with MS. .
Drug Discovery World
DECEMBER 18, 2023
University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. The post Gene therapy could reduce seizures in childhood epilepsy appeared first on Drug Discovery World (DDW).
Drug Discovery World
MAY 8, 2024
Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The post How can long-read nanopore sequencing support gene therapy delivery? appeared first on Drug Discovery World (DDW).
Drug Discovery World
SEPTEMBER 15, 2023
Advanced Therapies Europe took place from 9-12 September 2023 in Portugal. DDW’s Megan Thomas asked attendees: What is most important to scale from discovery to commercialisation of cell and gene therapies (CGTs)? Joel Eichmann, Co-Founder, Green Elephant Biotech It always comes down to time-to-market and risk minimisation.
Expert insights. Personalized for you.
284 
Let's personalize your content