Zemcelpro (dorocubicel)

P1/2, N=7, Recruiting, Ciusss de L'Est de l'Île de Montréal

In patients with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation (HSCT), transplantation with dorocubicel has been shown to result in neutrophil and platelet engraftment, as well as rapid T cell reconstitution. This article summarizes the key milestones in the development of dorocubicel leading to this first approval for the treatment of adult patients with haematological malignancies who require an allogeneic HSCT following myeloablative conditioning, for whom no other type of suitable donor cells is available.

Combining UM171-mediated expansion with adenine base editing (ABE), we precisely disrupted the P67 epitope of CD33, centered on phenylalanine 21, which is essential for gemtuzumab ozogamicin (GO) binding. Ultra-deep exome sequencing and GUIDE-seq revealed minimal off-target activity. Together, these findings establish CD33 P67 epitope-directed base editing as a precise, scalable strategy to generate immunotherapy-compatible, UM171-expanded CB grafts that safely enable GO integration into post-transplant care.

P2, N=30, Active, not recruiting, Ciusss de L'Est de l'Île de Montréal | Trial completion date: Oct 2027 --> Jun 2027 | Trial primary completion date: Oct 2024 --> Jun 2027

P1/2, N=12, Recruiting, ExCellThera inc. | Trial completion date: Jun 2026 --> Jun 2027

P2, N=15, Cordex Biolohics Inc

P1/2, N=12, Recruiting, ExCellThera inc. | Trial completion date: Dec 2025 --> Jun 2026 | Trial primary completion date: Dec 2023 --> Jun 2026

P2, N=30, Active, not recruiting, ExCellThera inc. | Recruiting --> Active, not recruiting | Trial completion date: Nov 2025 --> Feb 2026 | Trial primary completion date: Nov 2023 --> Feb 2026

P1/2, N=20, Active, not recruiting, Ciusss de L'Est de l'Île de Montréal | Trial primary completion date: Sep 2021 --> Oct 2023

P2, N=30, Active, not recruiting, Ciusss de L'Est de l'Île de Montréal | Recruiting --> Active, not recruiting | N=20 --> 30 | Trial completion date: Dec 2023 --> Oct 2027 | Trial primary completion date: Dec 2023 --> Oct 2024

All patients received tacrolimus and mycophenolate mofetil for GVHD prophylaxis. The results of 2 independent Phase II clinical studies demonstrated that infusion of a UM171 single CBT led to excellent clinical outcomes in patients with high and very-high risk malignancies including patients with p53+ mutation. We also observed encouraging results for patients undergoing second/third transplants (33% of the entire cohort). Clinical outcomes were similar between the two Transplant Centers.

P1/2, N=12, Recruiting, ExCellThera inc. | Trial completion date: Jul 2025 --> Dec 2025 | Trial primary completion date: Jul 2023 --> Dec 2023