FAQ

Act for Hope is an initiative that connects the global cell and gene therapy community, including patients, scientists, manufacturers, clinicians, and payers, to accelerate progress in advanced therapies. Our purpose is to close the gap between innovation and access by promoting collaboration, education, and shared understanding across the ecosystem. Through resources, events, and partnerships, we aim to make these life-changing therapies more available, affordable, and equitable for patients worldwide.

We work with patient advocates, scientists, clinicians, and policy experts around the globe to share knowledge, encourage collaboration, and shape sustainable pathways for advanced therapies.

We always welcome collaboration from organisations and individuals who share our goal of improving awareness, understanding, and accessibility of cell and gene therapies. Please get in touch with us at

Cell and gene therapies are advanced forms of treatment designed to address the root cause of disease rather than just its symptoms. They work by repairing, replacing, or modifying a patient’s genes or cells to restore normal function. Gene therapies introduce or correct genetic material within a person’s cells, while cell therapies use living cells, sometimes genetically modified, to repair or regenerate damaged tissues. Together, they hold the potential to offer long-term or even curative outcomes for conditions once considered untreatable.

Cell and gene therapies work by intervening at the biological source of disease. In gene therapy, scientists deliver new or corrected genetic material into a patient’s cells to fix or replace faulty instructions. In cell therapy, healthy or engineered cells are introduced into the body to restore normal tissue function or fight disease. These precise, targeted approaches enable the body to repair itself rather than relying on ongoing treatment of symptoms.

Manufacturing begins by collecting cells from a patient (autologous) or a donor (allogeneic). These cells are carefully transported to specialised GMP facilities, where they are modified to correct or enhance their function. The modified cells are then expanded, tested extensively for safety, purity, and potency, and cryopreserved until they are ready to be returned to the clinical site for patient infusion. Each step is tightly controlled to ensure product quality, consistency, and regulatory compliance.

Therapies are currently available for a limited number of conditions, mostly rare genetic disorders (e.g., haemoglobin disorders such as sickle cell disease or β-thalassemia) and some types of blood cancers. Clinical research is underway into other disease types (including solid tumours and autoimmune diseases), but most approved treatments remain in those more specialised areas. Eligibility depends on the specific therapy, the condition, disease stage, and patient health.

The cost varies significantly by treatment and location. For marketed gene and cell therapies in 2025, one-time treatments have listed prices often in the US $2 M to US $4.25 M range. That said, cost depends on region, healthcare system reimbursement, and whether outcomes-based payment models are used. Some health-systems negotiate differently, and out-of-pocket costs for patients depend on their insurance or national health coverage.

One of our key goals is to support scalability and affordability by promoting collaboration across the full ecosystem, from research and manufacturing to reimbursement. We work with regulators, payers, and industry partners to help shape policies that make these therapies more accessible, sustainable, and equitable for patients worldwide.