Brian Campbell, Vice President and General Manager of the US for Orchard Therapeutics, A Kyowa Kirin Company, discusses the critical role of newborn screening in identifying patients with metachromatic leukodystrophy (MLD), a rare and fatal neurometabolic disorder typically seen in children. Campbell also goes on to share his perspectives on the systemic changes needed across regulation, reimbursement, and infrastructure to ensure that transformative gene therapies can meaningfully reach patients.
Can you introduce Orchard Therapeutics, the mission behind the company, and the scientific approach it was built on?
Orchard was founded in 2015 by physician-scientists and gene therapy leaders, including our CEO and co-founder, Bobby Gaspar, M.D., Ph.D., with the goal of translating pioneering academic research in hematopoietic stem cell (HSC) gene therapy into medicines that could reach patients globally. In 2018, Orchard acquired GSK’s rare-disease gene therapy portfolio, adding programs for ADA-SCID, MLD, Wiskott-Aldrich syndrome and beta thalassemia.
Bobby’s motivation came directly from his experience caring for children with ADA-SCID and seeing firsthand what gene therapy could potentially achieve. By taking a patient’s own blood stem cells, inserting a working copy of the faulty gene, and returning those modified cells to the patient, HSC gene therapy has the potential to address the underlying cause of disease through a single treatment.
That vision became Orchard’s mission, and it is what motivated me to join the company: the opportunity to help translate transformative science into commercially available treatments for patients.
Can you give us an overview of metachromatic leukodystrophy (MLD) and explain why early detection in this disease is critical?
MLD is a rare, rapidly progressive, and irreversible genetic disease that affects the brain and nervous system and is ultimately fatal. Patients may appear healthy at birth, but age of first symptoms can range anywhere from birth to 17 years old. In late-infantile MLD, the most severe form, most children die within five years of symptom onset. The disease has a devastating impact on patients and their families.
Approximately 1 in 100,000 births in the US are affected by MLD, although we see significantly higher incidence rates in certain ethnic populations such as the Habbanite Jewish, Native Alaskans, and Navajo population.
Our focus is on early-onset MLD, which includes the late-infantile form, where symptoms typically begin before 30 months of age, and the early-juvenile form, where symptoms generally begin between 30 months and seven years of age.
It is difficult to diagnose children before symptom onset, in the small window where they may be eligible for treatment intervention. The central challenge is identifying these children before the irreversible neurologic damage has advanced.
Being a genetic condition, many families received an MLD diagnosis for their older child, because they showed symptoms, making them ineligible for possible intervention. This diagnosis then enabled early testing and diagnosis for their younger children. When asked about the impact MLD has on families, one mother said, “it is difficult to describe the pain of saving one child, while losing another to the same condition.”
That is why early diagnosis – and newborn screening – is so important. At Orchard, prioritizing education and early identification is central to our mission.
You can visit this website to learn more about MLD and an approved treatment option.
What would universal newborn screening mean for children and families affected by MLD?
Physicians and families face an extraordinarily difficult situation when a child is diagnosed after the treatment window has closed. At that point, care is generally focused on managing symptoms and providing supportive or palliative care.
Universal newborn screening is the most systematic way to identify children with MLD before symptoms emerge and while they may still be eligible for treatment intervention. Until MLD newborn screening is widely implemented, the diagnosis of a symptomatic child will continue to be what often prompts testing of younger siblings. Not a month goes by when I don’t hear about a family facing this situation. Too often, the older child is already symptomatic, while the younger sibling may be identified early enough to be eligible for intervention.
MLD was added to the federal Recommended Uniform Screening Panel, or RUSP, in December 2025 following sustained work by patient advocates, clinicians and public-health experts. That was an important milestone, but RUSP inclusion is a federal recommendation, not a mandate. Each state must complete its own legislative or administrative process, laboratory validation, funding, and implementation.
While that process takes time, the equity implications are immediate. In practical terms, a child’s opportunity for early diagnosis can depend on the U.S. state in which that child is born. That is a disparity in our healthcare system that I find very difficult to justify, especially when MLD screening is recommended at the federal level. At Orchard, we’re committed to supporting state-level implementation and education for local providers about screening and referral pathways in the event of a positive screen for MLD.
What policy changes are being considered to help states implement the RUSP more consistently and quickly?
Congress is considering the Newborn Screening Saves Lives Reauthorization Act of 2025, which would reauthorize federal newborn-screening programs and provide additional support for state capacity and infrastructure. As of July 2026, H.R. 4709 had been forwarded by the House Energy and Commerce Health Subcommittee to the full committee by voice vote.
Funding is an important barrier, but it is not the only one. States differ in their legislative and rulemaking processes, laboratory capacity, staffing, and implementation timelines.
For conditions such as MLD, without universal newborn screening, it is difficult to diagnose children before symptom onset, in the window where they may be eligible for treatment intervention.
Early identification is not just the right thing to do for patients and families, but also for the broader US healthcare system.
When you think about the current policy discussions in the US which are focused on pricing and innovation, do you think this may affect the long-term sustainability of innovation in rare diseases?
Absolutely. Policy choices can directly affect whether companies continue investing in rare and ultra-rare diseases. These programs face distinctive development and commercialization challenges because the patient populations are so small. Orphan-drug incentives help make sustained investment possible and should be preserved, with appropriate safeguards and accountability.
Over time, the technologies, manufacturing capabilities, and regulatory knowledge developed for rare diseases may also support programs in larger indications. As HSC gene therapy platforms mature and the number of potential applications grows, greater standardization, manufacturing experience and scale may improve efficiency and reduce portions of the cost structure.
Orchard has built a foundation spanning research, manufacturing, regulatory development, and commercialization across global markets. We are now applying that experience to improve efficiency and extend the potential of the platform.
What gives me confidence is the growing recognition across the system that speed and efficiency matter more than ever.
Where scientifically appropriate, well-validated biomarkers that are reasonably likely to predict clinical benefit may support accelerated development and approval, with confirmatory evidence and long-term follow-up generated after approval. Outcomes-based reimbursement can complement that approach by aligning payment with measurable performance over time.
Payers, manufacturers, regulators and researchers all need to bring the same spirit of innovation to the table. The science of rare diseases – particularly as cell and gene therapies continue to mature – is advancing rapidly. Now, the healthcare, regulatory and reimbursement systems that support these innovations need to keep pace.
What other systemic changes are needed to sustain innovation in rare and ultra-rare diseases?
The regulatory environment must evolve alongside the science.
Evidence requirements in ultra-rare, rapidly progressive diseases should be scientifically rigorous but proportionate to disease prevalence, natural history, urgency and the ethics of available comparators. Regulators have tools – including external controls, natural-history data, biomarkers and accelerated pathways – that may be appropriate when traditional randomized designs are infeasible or unethical. Regulatory innovation has supported important advances in other therapeutic areas, and the same willingness to use scientifically rigorous, fit-for-purpose pathways should be applied to rare and ultra-rare diseases.
When the total diagnosed population is extremely small, evidence expectations must account for what is feasible. That places a premium on robust natural-history data, clinically meaningful endpoints, biomarkers where validated, and long-term follow-up.
Looking forward, how does Orchard define its development focus, and will the company continue to focus on ultra-rare disease?
Neurometabolic and rare diseases remain our primary development focus for the foreseeable future. At the same time, we increasingly think about our future in terms of what the HSC gene therapy platform can do, rather than focusing on any single indication.
Our approach modifies a patient’s own blood stem cells outside the body by inserting a working copy of the missing or faulty gene. After those cells are returned and engraft in the bone marrow, they can self-renew and produce multiple blood-cell lineages. Some descendant cells can migrate into tissues, including across the blood-brain barrier, creating the potential to deliver therapeutic proteins to the central nervous system and other organs.
That platform may have applications beyond ultra-rare neurometabolic disease, but any expansion must be driven by strong biological rationale, significant unmet need, and evidence that HSC gene therapy offers a meaningful advantage.
Another important area of innovation is conditioning. Current HSC gene therapy requires conditioning to create space in the bone marrow for the modified cells to engraft. Developing less intensive conditioning approaches while maintaining durable engraftment could improve the patient experience and broaden the potential applicability of the platform.
What is the message you want to leave with about what Orchard is building in gene therapy, and what does the US need to do to ensure these kinds of treatments can fulfil their promise for the patients still waiting?
At Orchard, we have built the infrastructure and demonstrated what it takes to move HSC gene therapy from the laboratory to patients. Strong science is essential, but it is only part of the equation. Manufacturing, regulatory approval, reimbursement, treatment-center readiness, and patient identification all must work together – and each presenting its own challenges and costs.
We must think about access from the earliest stages of clinical development. We don’t want to spend years developing a therapy, at significant cost, only to find that patients cannot access it. That helps nobody, and it gives families false hope along the way. So, we are working to demonstrate that it is possible to build the commercial, regulatory, access, and advocacy infrastructure needed to deliver equitable access and commercial viability alongside innovative science. That has been part of our program design from the beginning.
More broadly, the entire ecosystem needs to evolve for gene therapies to reach their potential. As a society, we expect clinicians and researchers to be innovative. If we hold that standard for science, we must hold the same standard for regulators, payers, and the commercial infrastructure around these therapies.
Innovation cannot stop at the laboratory door. It must extend to how therapies are developed and approved, how they are reimbursed and how eligible patients are identified and treated.
If we can move all those pieces forward together, gene therapy may become a viable option for the patients who are still waiting.
