Clinical Trials
Multi-jurisdictional translational studies targeting critical unmet medical needs. 2026 / 2027 Pipeline.
Across all protocols, the distinction between established and investigational care is maintained explicitly. Every entry provides full scientific and regulatory specifications alongside a companion plain-language summary for patients and families. The companion plain-language summaries include explicit statements on the limits of the claim — ensuring clarity for patients and regulatory bodies alike.
EXPLORE INVESTIGATIONAL PROTOCOLS BY AUDIENCE PERSPECTIVE
Clinical Protocols & Plain-Language Summaries
Standing disclaimer: The descriptions below represent clinical trial protocols in active preparation or regulatory review. None of these investigational therapies have been approved by regulatory agencies as established treatments.
Alzheimer's Disease and Related Dementias
A Phase 1, Open-Label, Dose-Escalation Study of AAV-Mediated Neurotrophic Gene Transfer in Early Symptomatic Alzheimer's Disease
Supporting the brain's own repair signals
Brain cells rely on a set of natural growth factors to stay healthy and stay connected to one another. In Alzheimer's disease, that supply falls away years before memory problems become noticeable, and the connections between cells are lost along with it. This study tests a one-time treatment that carries instructions into the brain so it produces more of these growth factors on its own, instead of requiring repeated infusions of a drug. The first purpose of the study is to establish safety. Alongside that, we track markers in blood and spinal fluid, brain scans, and standard memory testing to see whether the treatment is reaching and affecting the intended cells.
Sarcopenia and Age-Related Muscle Loss
A Phase 1/2a Study of Intramuscular AAV9-Follistatin-344 in Adults with Age-Related Sarcopenia
Releasing the brakes on muscle
The body makes proteins whose job is to limit muscle growth. They are useful in early life and unhelpful in later life, when muscle is already being lost faster than it is rebuilt. Follistatin is the body's natural counterweight to those proteins. This study delivers instructions for extra follistatin directly into specific muscle groups, in a single session of injections, so those muscles produce it locally rather than exposing the whole body to it. Success is measured by what the muscle can do — strength, standing from a chair, walking speed — alongside imaging of the muscles treated.
Thymic Regeneration and Immune Reconstitution
A Phase 1 Study of Image-Guided Intrathymic Vector Administration for Restoration of Thymic Output in Adult Immunosenescence
Restarting the gland that trains the immune system
The thymus is a small gland behind the breastbone that trains new immune cells and teaches them to leave the body's own tissue alone. It begins shrinking after puberty and is largely inactive by middle age, which is one reason infections become more dangerous and autoimmune problems more common with age. Reaching this gland has been the obstacle, not knowing what to send it. This study delivers a low dose of treatment directly into the thymus under image guidance, and the treatment is built with a control that allows it to be turned up, turned down, or switched off after it is given.
Sensorineural Hearing Loss
A Phase 1/2 Study of Intracochlear Gene Transfer in Adults with Sensorineural Hearing Loss
Treating the ear rather than amplifying sound
Most permanent hearing loss comes from damage to the sensory cells of the inner ear. Those cells do not grow back. Hearing aids and implants work around the damage by making sound louder or bypassing the ear entirely, but neither repairs the part that failed. The inner ear is a small, enclosed, fluid-filled space, which makes it one of the more workable targets in the body for this kind of treatment: a very small dose can be placed directly where it is needed. This study treats one ear, leaving the other unchanged, and measures both hearing thresholds and the ability to follow speech in a noisy room.
Inherited Retinal Disease and Blindness
A Phase 1/2 Study of Subretinal Gene Transfer in Adults with Inherited Retinal Degeneration
Delivering a working copy of a faulty gene
In inherited retinal disease, a single faulty gene causes the light-sensing cells at the back of the eye to fail over time. This study places a working copy of that gene beneath the retina in a single procedure. The eye is a favourable place for this kind of treatment: it is enclosed, it needs only a tiny dose, and the results can be watched directly with high-resolution scans in the clinic. Participants need to still have living light-sensing cells for the treatment to act on, so genetic testing and retinal scans determine who can take part.
Substance Use Disorder
A Phase 1 Study of Gene-Based Modulation of Reward Circuitry in Treatment-Refractory Substance Use Disorder
Targeting why relapse persists
Detox addresses the first days. The far harder problem is that the brain circuits involved in craving stay changed for years afterwards, which is why relapse remains common long after someone has stopped. Existing medications help some people and work by occupying receptors or making use unpleasant; none of them address the underlying change. This study is an early safety study of a treatment aimed at that persistence. It is open only to adults who have already tried approved medication and structured treatment programmes without lasting benefit.
Refractory Autoimmune Disease
A Phase 1 Study of AIRE-Directed Intrathymic Gene Transfer for Restoration of Central Tolerance, with Type 1 Diabetes as Index Indication
Correcting the filter, not just the damage
In autoimmune disease, the immune system attacks the body's own tissue. Most current treatment works by suppressing the immune system after that has already started. The thymus is where immune cells are normally screened and the self-attacking ones removed, and that screening weakens with age. This study delivers treatment directly to the thymus to restore that screening step, studied first in people recently diagnosed with type 1 diabetes, where the target is clearly defined and the remaining insulin-producing capacity can be measured accurately.
Inquire About Trial Participation
Patients, caregivers, and referring physicians may submit medical summaries for preliminary eligibility and inclusion criteria review.