Roundtable Part 2: What medical conditions currently benefit the most from cell and gene therapy? Do you see this list growing in the future?

In the past five years, the predominant indication has been in oncology leveraging chimeric antigen receptor somatic cells, but the prospect of regenerative and curative therapies using stem cells for treatment of degenerative disorders may be some of the most exciting in the future for those that have struggled to live with debilitating illness.

It has been exciting to see our partners in biopharmaceuticals developing medicines to treat rare diseases, in many forms. It is really difficult to pin point one specific condition. So many families have been affected by cancer, by blood disorders or autoimmune disease. For these families and patients – these advances in the treatments has given them hope and a pathway for their future.

We work to cure disease states that have not had the cure rate to date that potentially some of the new technologies will bring to the table. At the end of the day, we all need to keep new technologies and the research associated with them in mind, but we can never take our eyes off that we work for patients.

We are living in a time of unsurpassed scientific and technological progress that is generating unprecedented interest in the development of treatments that address unmet medical needs – including important advances in rare disease product development. To date, FDA has approved 10 gene therapy products covering certain acquired hematologic cancers (acute lymphoid leukemia and non-Hodgkin lymphomas) and hereditary disorders, including a rare form of blindness; the hereditary disease spinal muscular atrophy, type 1; beta-thalassemia, and the metabolic disorder adrenal leukodystrophy. Each of the therapies noted treats serious and life-threatening diseases or conditions – many of which are rare.

Oncology is still the dominant indication – with therapies for blood cancers still leading the way, however, promising new candidates for solid tumors are emerging and showing positive data in clinical trials. The majority of drugs in development (and currently approved) are autologous and the total number of patients treated still relatively low – but when allogeneic therapies become approved, volumes will increase dramatically. In addition, we are already seeing existing, approved, cell and gene therapies gaining additional approvals for the earlier use in treatment regimens – rather than third or fourth line. They are now being used as second line treatments and may soon become first line medication for some indications.

‎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.