George Buchman, Ph.D.
Research Fellow, Product Development, Catalent Cell and Gene Therapy
Gene therapies that make use of adeno-associated virus (AAV) vectors have been used to treat a number of monogenic diseases, many of which are extremely rare. Some of the most prevalent include cystic fibrosis, hemophilia, muscular dystrophy, sickle cell anemia, Huntington’s, Parkinson’s, Gaucher’s disease and spinal muscular atrophy. To date, cell therapies have also been used to treat blood cancers, including multiple myeloma and lymphoma.
Given the huge potential of these therapies, it is nearly assured that we will see this list grow over the coming years. There is currently a rich pipeline of hundreds of candidates in all phases of development.
With improvements to development and manufacturing processes, and strides in vector design, these therapies will become more accessible and realize their potential to markedly improve the lives of many more people.