Gene therapy has matured into a clinically validated modality, yet its path to patients remains uneven. In this conversation, Sander Van Deventer draws on decades of experience across academia, biotech founding, investment, and regulatory engagement. His perspective is grounded in concrete episodes, from early antibody development to the approval struggles of Glybera and the recent dosing of an ALS patient. For developers working at the intersection of science, manufacturing, and policy, his reflections offer a field-tested view of where programs succeed, where they fail, and what tends to be underestimated along the way.
The Narrow Safety Window
For Sander, the first hurdle in moving from lab to clinic is safety. He argues that parts of the field have underestimated how small the therapeutic window can be.
“The safety window is very small, and fatalities are bound to happen even though they shouldn’t happen.”
In preclinical work, much can look robust. Biodistribution studies may suggest that a vector behaves as intended. But translation remains the inflection point.
“Everything can work in a mouse, but translational steps need to be in place.”
If you are preparing an IND, that distinction is not academic. It is the difference between a clean early trial and a program that stalls at the first sign of toxicity.
Sander stresses that biodistribution must be fully understood before moving forward. In gene therapy, systemic exposure, off target expression, and immune responses are not theoretical risks. They are design constraints. His own development history, he notes, has been marked by caution in this respect.
Manufacturing as a Decisive Variable
Enthusiasm for innovation does not blind him to operational reality. He remains deeply interested in CAR T, including in vivo approaches, but questions whether current manufacturing models are sustainable.
“If the therapies cost 150,000 euros per patient, then it’s a lost case.”
That statement reflects not only pricing pressure but also the structural limits of bespoke production. If you are building a cell therapy company, cost of goods is not an afterthought. It shapes access, reimbursement, and political viability.
Sander argues that scaling manufacturing early is essential to reducing costs. The example of haemophilia B patients who remain years free of disease after treatment shows what durable benefit can look like. But durability alone does not solve accessibility. Convenience and broad availability, in his view, should be part of the design brief from the beginning.
About Sander van Deventer
Sander Van Deventer trained as a physician and held a professor title at both Academic Medical Center Amsterdam Experimental Medicine & Gastroenterology and Hepatology) and Leiden University Medical Center (Translational Gastroenterology). He was previously a scientist at Rockefeller University, where he contributed to the discovery of Remicade in the 1980s and worked on multiple antibody programs. He founded uniQure, led the development of Glybera, the first gene therapy to be approved in the Western World, Hemgenix and AMT-130 and later founded a gene therapy company VectorY, with Forbion as co-founder and led the development of its compounds, bringing VTx-002 for ALS into the clinic. For over a year, he served as CSO of a Chinese firm Porton Biologics, where he helped shape regulatory frameworks and build a gene therapy platform.
Glybera and the Politics of Approval
The most significant setback of his career was Glybera, the first gene therapy approved in Europe. Clinically, it worked. Regulatory endorsement came from one committee, yet other agencies voted it down twice. The company’s valuation collapsed from 150 million to 10 million before being reinvented and eventually securing approval.
The experience reshaped his assumptions. “I believed much more that data would convince people.” Instead, he saw how political dynamics, financial considerations, and regulatory caution could override scientific merit.
Glybera’s price, around 800,000 euros, was often cited as prohibitive. Van Deventer argues that such debates must consider lifetime healthcare savings, not just upfront cost. Yet he is clear-eyed about the consequences. Politicians and insurers, he says, can effectively block access. For young developers, that reality means engaging governments early and building relationships beyond scientific advisory boards.
About VectorY
Based in Amsterdam, VectorY is developing gene therapies for neurodegenerative diseases. Its platform uses adeno-associated virus (AAV) vectors to deliver the genetic code of therapeutic antibodies directly into the nervous system, allowing cells to produce the antibodies themselves. The company’s lead program targets TDP-43 pathology in amyotrophic lateral sclerosis (ALS), a molecular driver present in most cases of the disease.
VectorY
2020
Amsterdam, The Netherlands
AAV gene therapies for neurodegenerative diseases
Getting Drugs to Market, Not Just to Publication
Asked what young developers should learn from failure, Van Deventer focuses less on technical lessons and more on orientation.
“I don’t care about publications or prizes; I care about drugs getting to market.”
That stance affects how he evaluates programs. In investment settings, he observes founders spending 45 minutes on data when pitching projects to Forbion. Science, in his view, should be assumed as a baseline. What matters equally are regulatory strategy, manufacturing plans, and reimbursement pathways. If you are presenting to investors or partners, an elegant dataset will not compensate for a thin plan on access.
His leadership lesson is similarly pragmatic. “Always recruit people that are smarter than yourself.” For him, that requires recognizing one’s own blind spots and building teams accordingly. In a field where biology, regulation, and finance intersect, humility becomes a strategic asset.
He also emphasizes international perspective. The first ALS patient for his current program was treated in the United States months ahead of Europe. China, he notes, is now one of the fastest growing markets in cell and gene therapy. Speaking only to local stakeholders is no longer sufficient.
VectorY and the Move Into the Clinic
Sander is currently President of R&D at VectorY, a gene therapy company developing treatments for ALS, Huntington’s disease and other neurodegenerative disorders. A first patient was dosed in February 2026, marking a transition from preclinical ambition to clinical execution. The program reflects his continued focus on neurological disease, where delivery, biodistribution, and safety margins are tightly linked.
The step into patients is significant not only scientifically but operationally. As he has emphasized throughout his career, early clinical progress depends on groundwork laid years earlier in vector design, regulatory planning, and manufacturing readiness. The ALS program brings together those elements in a setting where timelines and patient expectations are unforgiving.
Forbion and the Broader Investment Lens
Van Deventer is also a co-founder of Forbion, a life sciences investment firm active across venture development and venture investment, from seed stage through growth. His role has given him a vantage point beyond a single program or platform.
At Forbion, he sees how companies present themselves and where they struggle. Scientific depth is expected. What differentiates teams is their understanding of regulatory positioning, reimbursement strategy, and execution risk. That broader lens reinforces his central argument: bringing a therapy to market requires fluency not only in biology, but in the systems that surround it.
Why Sander’s Keynote Matters
Sander Van Deventer’s keynote brings together a perspective that is rare in cell and gene therapy. He has moved from academic medicine to antibody discovery, from founding and building gene therapy companies to navigating European regulatory setbacks, and from shaping regulatory frameworks in China to dosing patients with a new ALS therapy. Few speakers can draw on that breadth of direct experience across science, company building, investment, and policy.
What makes his contribution particularly relevant now is the consistency of the lessons across those contexts. He has seen a drug work clinically and still struggle to reach patients. He has watched valuations collapse despite regulatory endorsement. He has helped construct platforms in emerging markets while confronting the realities of reimbursement negotiations in Europe. That combination allows him to speak not only about what advances a program scientifically, but what determines whether it survives commercially and politically.
