Jaap Jan Boelens
Chief Medical Officer, ISCT
Jaap Jan Boelens, MD, PhD, is Chief Medical Officer of the International Society for Cell and Gene Therapy (ISCT). A clinician who came up through bone marrow transplant, he was pulled into advanced therapies more than two decades ago and now treats the patients living inside the access gap he describes.
Boelens sees cell and gene therapy from the chair across from the patient. He treats sickle cell patients weighing a gene therapy they qualify for against a standard transplant, and he has watched single-case agreements with payers drag on so long that patients back out of the newer therapy entirely. He is a proponent of the long-term, real-world monitoring these products demand, noting that while the field has five decades of data on bone marrow transplant, it has only a few years on many ex vivo gene therapies, for patients who may need the result to hold for decades.
Boelens appeared on Open Door Salon alongside Bambi Grilley, President-Elect of ISCT, for a conversation with host Lori Ellis on why patients walk away from cures they qualify for. He explained why the contracting and single-case-agreement phase is its own barrier to access, why in vivo gene therapy for genetic disease is likely more than a decade away, and what worries him most about what we do not know regarding cell and gene therapy activity in China.
Frequently Asked Questions About Jaap Jan Boelens
Who is Jaap Jan Boelens?
Jaap Jan Boelens, MD, PhD, is Chief Medical Officer of the International Society for Cell and Gene Therapy (ISCT). A clinician who came up through bone marrow transplant, he was pulled into advanced therapies more than two decades ago. He treats the patients living inside the access gap he describes.
What patients does Jaap Jan Boelens treat?
Jaap Jan Boelens treats sickle cell patients who are weighing a gene therapy they qualify for against a standard bone marrow transplant. He sees cell and gene therapy from the chair across from the patient, and he came up through bone marrow transplant. He has spent more than two decades in advanced therapies.
Why does Jaap Jan Boelens say patients walk away?
Jaap Jan Boelens argues that the contracting and single-case-agreement phase is its own barrier to access. He has watched those agreements drag on so long that patients back out of the newer therapy entirely. On Open Door Salon he said: “I've seen patients backing out on the gene therapy because the track to get a single-case agreement in place is going to take too long.”
What does Jaap Jan Boelens argue about long-term monitoring?
Jaap Jan Boelens is a proponent of the long-term, real-world monitoring these products demand. The field holds five decades of data on bone marrow transplant and only a few years on many ex vivo gene therapies, for patients who may need the result to hold for decades. On Open Door Salon he said: “For the bone marrow transplant, we have five decades of experience, but for the cell and gene therapies, we don't know. These patients need to live another five, six decades.”
When does Jaap Jan Boelens expect in vivo gene therapy?
Jaap Jan Boelens places in vivo gene therapy for genetic disease more than a decade out, and he points to dosing as an open issue. On Open Door Salon he said: “The in vivo gene therapy is likely more than a decade away, and then it's an issue with the dosing: how much to give to get sufficient cells in vivo and not overload the system.”
On Open Door Salon
“Why Patients Walk Away From a Cure They Qualify For”
Bambi Grilley & Jaap Jan Boelens · July 15, 2026
Episode page & show notes on Open Door Salon
In this episode
- Why the contracting phase delays patient access
- Single-case agreements and patients who walk away
- Real-world monitoring and the five-decade question
- How far away in vivo gene therapy really is
- What we don't know about China
- What inspired a career in advanced therapies
Topics
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