GOSH has treated in June 2015 a young leukemia patient under a special license from the Medicines & Healthcare products Regulatory Agency (MHRA) with Cellectis’ TALEN® gene edited allogeneic UCART19 product candidate because no other therapies were available for refractory relapsed Acute Lymphoblastic Leukemia (ALL) following mismatched allogeneic stem cell transplantation.
In response to an unsolicited request from Professor Waseem Qasim, Consultant Immunologist at GOSH and Professor of Cell and Gene Therapy at University College London (UCL) Institute of Child Health, Cellectis gave its approval for the use of its UCART19 product candidate and technologies under GOSH’s "Specials" license and responsibility, for the particular clinical needs of that individual patient.
Professor Qasim says: “The successful treatment of a patient with UCART19 cells represents a landmark in the use of new gene engineering technology. If replicated in other patients, it could represent a huge step forward in treating leukaemia and other cancers.”
“We are very glad for this young patient to have benefited from our highly innovative TALEN® gene edited allogeneic CAR T therapy UCART19. We expect to accelerate our clinical development of TALEN® gene-edited allogeneic CAR-T therapies to further confirm this encouraging clinical proof of concept,” said Doctor Mathieu Simon, MD, Executive Vice President, Chief Operating Officer at Cellectis.
“Our team aims to provide to patients, with unmet medical needs, access to the first allogeneic CAR-T therapy, UCART19 made with Cellectis’ TALEN® gene-editing technologies,” said Doctor André Choulika, Founder, Chairman and Chief Executive Officer of Cellectis. “Cellectis had, is and will invest significant amounts of energy and creativity to provide cancer patients with an accessible, cost-effective, off-the-shelf allogeneic CAR-T therapies across all geographies. UCART19 has been provided for to a patientwho could not undergo an autologousCAR-T therapy. Our goal is to make our product candidates accessible to anyone.”
First Clinical Application of Talen Engineered Universal CAR19 T Cells in B-ALL
Gene Therapy and Transfer
Program: Oral and Poster Abstracts
Session: 801. Gene Therapy and Transfer:
Poster I Saturday, December 5, 2015, 5:30 PM-7:30 PM
Hall A, Level 2 (Orange County Convention Center)
Waseem Qasim, MBBS PHD1*, Persis Jal Amrolia2*, Sujith Samarasinghe, MD, PhD3*, Sara Ghorashian, MD, PhD, FRCPath1*, Hong Zhan, PhD4*, Sian Stafford, PHD1*, Katie Butler, PHD1*, Gul Ahsan5*, Kimberly Gilmour5*, Stuart Adams, PHD5*, Danielle Pinner5*, Robert Chiesa5*, Steve Chatters, PHD5*, Sue Swift, PHD1*, Nicholas Goulden, MD, PhD3, Karl Peggs, MBBChir, MRCP, FRCPath6*, Adrian J Thrasher, MD, PhD1*, Paul Veys2* and Martin Pule, PhD7*
1 INSTITUTE OF CHILD HEALTH, UCL, London, United Kingdom
2 Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, United Kingdom
3 Department of Haematology, Great Ormond Street Hospital For Children, London, United Kingdom
4 Institute of Child Health / Molecular and Cellular Immunology Unit, University College London, London, United Kingdom
5 GREAT ORMOND STREET HOSPITAL, London, United Kingdom
6 CANCER INSTITUTE, UCL, London, United Kingdom
7 Cancer Institute, University College London, London, United Kingdom
UCART19 is a potential best-in-class allogeneic engineered T-cell product for treatment of CD19 expressing hematologic malignancies, initially developed in Chronic lymphocytic leukemia (CLL) and Acute lymphoblastic leukemia (ALL). Servier has an option under the collaboration agreement to acquire the exclusive rights to further develop and commercialize UCART19. Engineered allogeneic CD19 T-cells currently stand out as a real therapeutic innovation for treating various types of leukemia and lymphoma. Cellectis’ approach with UCART19 is based on the preliminary positive results from clinical trials using products based on the CAR technology and has the potential to overcome the limitation of the autologous current approach by providing an allogeneic frozen, “off the shelf” T-cell based medicinal product.
About Great Ormond Street Hospital (GOSH)
Great Ormond Street Hospital for Children NHS Trust is the country’s leading centre for treating sick children, with the widest range of specialists under one roof. With the UCL Institute of Child Health, they are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.
About the UCL department of hematology
The UCL department of hematology is the major tertiary referral center in the UK for all types of hematological malignancies. They have assumed a global leadership position in stem cell transplantation and adoptive cell therapy for leukemia patients.