Gene Therapy in Haemoglobinopathies Study Day

This 1‑day study day will explore the full multidisciplinary pathway for gene therapy in haemoglobinopathies.

UCLH is a leading centre for gene therapy delivery in the south of the UK, with the first patient in the country receiving the revolutionary gene‑editing treatment Casgevy here. This study day is designed to share our experience, showcase our capabilities, and support referring centres and teams developing their own gene therapy pathways.

A breakthrough for Beta Thalassaemia: The First Patient in the South of the UK Treated at UCLH

In 2025, a UCLH patient, Kavita Mehta, became the first person in the south of the UK to receive the new gene‑editing therapy Casgevy.

Kavita had relied on blood transfusions every 3–4 weeks since she was a baby. The constant cycle of transfusions and iron chelation shaped every part of her life — school, travel, work, and day‑to‑day routines.

After receiving four phials of Casgevy in November, she spent six weeks recovering at UCLH as her body began producing normal red blood cells for the first time since infancy. She was discharged just before Christmas — her first ever Christmas not planned around a transfusion schedule.

As UCLH haematology consultant Dr Ben Carpenter explains:

“We are pleased with the way things are going for our first patient… We are now seeing normal red blood cells being produced by her own bone marrow for the first time since she was born.”

This represents a potential cure for many patients — without donor shortages, rejection risks, or transplant‑related complications.

Who should attend this study day?

This session is aimed at:

• Referring centres seeking to understand eligibility, pathways, and referral processes

• Teams planning to establish a gene therapy service, including setup considerations, governance, and capacity

• Red‑cell teams looking to update knowledge on rapidly evolving gene‑therapy options

• Clinical leaders and managers wanting to understand the wider implications for service design and demand

What will be covered?

This 1‑day study day will explore the full multidisciplinary pathway for gene therapy in haemoglobinopathies, including:

• Introduction and overview of gene therapy and haemoglobinopathies

• Red‑cell pathways: patient selection, clinical pathways, and decision‑making

• Red‑cell CNS perspective: educational needs and psychosocial considerations

• Gene therapy pathway: clinical trials, eligibility, outcomes, and rationale

• Cellular therapy nursing role: coordination and patient support across the pathway

• Apheresis and laboratory processes: ensuring high‑quality stem‑cell collection

• Inpatient care: busulfan sampling, monitoring, and safe delivery of therapy

• Ph*rm*cy role: ensuring safe preparation, handling, and administration of gene therapy

• Fertility considerations: reproductive counselling and pathway planning

• Psychological support: psychosocial frameworks for patient wellbeing

• Panel discussion: future directions and opportunities

Register today before places sell out!

Info: Allogeneic HSCT, Clinical Trials, or Gene Therapy

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