ATMP Related Case Studies

HOPE-B Gene Therapy Trial for Haemophilia B

In 2019 working with Professor Niamh O’Connell, Consultant Haematologist at St James Hospital, three patients with Haemophilia B were enrolled in a gene therapy trial. A license for GMO release was secured from the Environmental Protection Agency. Following six months of lead-in visits, in early 2020 the three patients were administered the AAV5-hFIXco-Padua therapeutic agent. Each patient received a single dose of gene therapy and attended follow-up visits in the CRF following their treatment. Thus far, all three patients have had excellent outcomes.

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CARTITUDE-1 CART-T Trial for Multiple Myeloma

In 2023, working with Professor Larry Bacon, Clinical Lead for the National Adult CAR-T Centre at St James Hospital, the first CAR-T trial is performed in Ireland. The multicentre global is seeking to evaluate the efficacy and safety of CAR-T as a frontline therapy in people newly diagnosed with multiple myeloma, a rare form of blood cancer. The study is recruiting at present.

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BIIB078 Trial for Amyotrophic Lateral Sclerosis

In 2020, working with Professor Orla Hardiman of Trinity College Dublin, the SJH-CRF received pre-approval as a Phase 1 unit to conduct a ‘Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis, (Biogen).

The ACU compounding capacity was used in conjunction with our in-patient facilities to provide access to a Phase 1 clinical trial of an antisense oligonucleotide investigational product for the treatment of a rare aggressive genetic variant of Amyotrophic Lateral Sclerosis. The patient was one of just 27 worldwide to be able to avail of the treatment which involved repeated intrathecal injections as well as an overnight stay. The SJH-CRF has since recruited a number of patients to similar Phase 1 studies for ALS.

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