Dr. Jayson Stoffman

Dr. Stoffman completed his subspecialty residency training in Winnipeg, Canada as a Pediatric Hematologist/Oncologist with a Thrombosis/Hemostasis fellowship in 2005. He is an Associate Professor in the Dept. of Pediatrics and Child Health, and the Program Director for General Pediatrics at the University of Manitoba. His clinical interests are in coagulation disorders and hemoglobinopathies in children, where he is also very involved in education and advocacy. Dr. Stoffman is a previous Chair of the World Federation of Hemophilia Twinning Committee, and led a successful twinning partnership in Delhi, India. He is also a past President and Privacy Officer of the Association of Hemophilia Clinic Directors of Canada and was the inaugural Chair of the Medical and Research Advisory Board of the Sickle Cell Disease Association of Canada. Currently, he is the Chair of the Health Care Providers Working Group of GANSID and the medical advisor for the Sickle Circle of Manitoba.

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Dr. Jennifer Bryan

Dr. Jennifer Bryan, Emergency Physician and Emergency Research Director at the University Health Network, is an Associate Professor at the University of Toronto. She led the inaugural national recommendations on addressing racism and colonialism in emergency medicine. She champions equity as Chair of the Canadian Association of Emergency Physicians Antiracism and Anticolonialism Committee. Dr. Bryan founded and chairs the UHN emergency department sickle cell working group, recognized nationally for their work in advancing timely pain relief for patients with sickle cell disease.

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Dr. Titilope Adeyemo

Dr. Titilope A. Adeyemo is a Professor of Hematology & Blood Transfusion in the Department of Hematology & Blood Transfusion, faculty of Clinical Sciences, College of Medicine, University of Lagos & a Consultant-Specialist hematologist to the Lagos University Teaching Hospital. She obtained her medical degree at the College of Medicine, University of Lagos, and had residency training in hematology & blood transfusion at the Lagos University Teaching Hospital earning a fellowship of the National Postgraduate Medical College of Nigeria (FMCPath). She also obtained a Master’s degree in Hemoglobinopathies from the University College London with a distinction. Her research and clinical interests are in benign blood diseases; sickle cell disease, inherited bleeding disorder (hemophilia), clotting disorders and blood transfusion. She is the current head of department of haematology & blood transfusion, Lagos University Teaching Hospital, deputy director, clinical trials at the Centre for Clinical Trials, Research and Implementation Science (CCTRIS), College of Medicine, University of Lagos and is principal investigator/co-investigator for several ongoing research studies including low dose aspirin for preventing intrauterine growth restriction and preeclampsia in sickle cell pregnancy (PIP-SICKLE): a randomized controlled trial, and Assessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell Disease (ACHiEvE-SCD). Prof. Titi Adeyemo has authored and co-authored over 100 publications in peer reviewed Journals.

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Shipra Kaicker, MD

I am a pediatric hematologist-oncologist and full-time teaching faculty at Weill Cornell Medical Center. I have practiced clinical medicine and taught and mentored medical students, residents, and fellows for over 20 years. I am particularly interested in global health and in improving the care of children and adults with hematological and oncological disorders in poorly resourced settings

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Dr. Yee Yee Yap

Dr Yap Yee Yee is a clinical haematologist who is looking after thalassaemia patients in Ampang Hospital. She had done her fellowship in haemostasis & thrombosis in Perth, Australia. She acquired her FRCPath(UK) qualification from UK in year 2022. She is currently the president of Malaysian Society of Patient Blood Management.

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Dr. Annette Akinsete

Dr. Annette Ofunne Akinsete has been the CEO/National Director of Sickle Cell Foundation Nigeria for several years, where she continues to propel successful innovative strategies for the prevention, management, care and support, as well as cure for sickle cell disorder through – inter alia – Public Private Partnerships, Arts in Medicine initiatives and Family Support Systems. As Co-Chair of the Top Management Committee of the Sickle Cell Foundation Nigeria/LUTH BMT (Bone Marrow Transplant) Programme (the first comprehensive BMT Programme for sickle cell disease in Africa), Akinsete provides the required critical, institutional oversight for the initiative. Under her watch, the first couple of transplant procedures have commenced. Annette Akinsete is a woman of many parts; she is – inter alia – a Consultant Public Health Physician, an astute Administrator, a Teacher and a Broadcaster. A distinguished Fellow of the National Postgraduate Medical College of Nigeria, she was Director in the Public Health Department of the Federal Ministry of Health for many years, representing the Federal Government of Nigeria at several multi-lateral and bilateral engagements all over the world. As a Public Health Physician, Akinsete has worked in every level of government in Nigeria – from local, State to Federal levels. At global level, she has contributed to World Health Organisation (WHO) publications on Non-Communicable Diseases (NCDs), served as WHO Fellow in Geneva and as UN Cares Facilitator at the United Nations HQ in New York. Dr. Akinsete, who is a member of several professional bodies and a Commissioner of the Nigeria NCDI Poverty Commission, is also an Adjunct Lecturer at the Eko University of Medicine, a member of the Board of the University of Ibadan Research Foundation, an Executive of NCD Alliance Nigeria, and a Global Facilitator of GANSID (Global Action Network for Sickle Cell & other Inherited Diseases). She is a key resource person for the West African Health Organisation and represents Women’s Board at the United Nations. A Chartered Management Consultant, a Certified Genetic Counsellor and a recipient of numerous awards, Akinsete continues to be involved in the formation of young girls as a Brownie Owl. She has been happily married to Captain Sina Akinsete for many years.

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Dr. Simpson

Dr. Simpson received her medical degree from McGill University and completed her General Pediatrics training and Hematology/Oncology fellowship at the Hospital for Sick Children and the University of Toronto. During her fellowship training, she also completed a Master’s in Public Health at the Johns Hopkins Bloomberg School of Public Health as a Global Health Scholar. Dr. Simpson is a pediatric hematologist/oncologist in the Division of Hematology/Oncology at CHEO and she is also appointed as an Associate Professor in the Department of Pediatrics at the University of Ottawa Faculty of Medicine. Her major clinical and research interests have centered upon health systems improvement for children and adolescents with sickle cell disease and other chronic hematologic disorders. From July 2021 to August 2024, Dr. Simpson served as Assistant Dean for the Office of Equity, Diversity and Inclusion for the University of Ottawa Faculty of Medicine. In August 2024, she assumed the role of Division Head for CHEO Hematology/Oncology. As Division Head, Dr. Simpson looks forward to opportunities to represent the interests of oncology patients and families, to advance healthy equity and innovation, and to advocate for superior systems of care for pediatric oncology at local, regional levels and national levels.

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Josephine Sirna

With over 50 years living with transfusion-dependent thalassemia, Josephine has used her lived experience as a patient to raise awareness, educate and advocate on behalf of patients with thalassemia and other inherited blood disorders. She is a long-time volunteer in the thalassemia community, who led the successful effort to include thalassemia patients in Canada’s Hepatitis C compensation plan. She was an inaugural member of Canadian Blood Services’ community liaison committee. Josephine continues her volunteer work as a board member of the Thalassemia Foundation of Canada and the Network of Rare Blood Disorders while working full time for the federal government. Josephine obtained her B.Sc. in Biochemistry from McMaster University and her M.Sc. in Laboratory Medicine and Pathobiology from University of Toronto. She resides in Ottawa, Canada’s capital, with her husband and three young adult children.

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Bessie Calabria

Bessie Calabria is a thriving beta thalassemia major patient who is the President of the Thalassemia Foundation of Canada and board member of Thalassemia International Federation. She is passionate about patient advocacy and education and believes that knowledge is a powerful way to improve patient outcomes. She is a graduate of Queens University and has worked in leadership roles in the financial services industry for 25+ years. Most of all, she loves spending time with her family and friends and thalassemia community.

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Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY® (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

September 26, 2024: The GANSID and its members in Canada serving the sickle cell and thalassemia communities are pleased with the news of CASGEVY receiving marketing authorization for the treatment of these two disorders. CASGEVY is Canada's first CRISPR-based gene-editing therapy to receive Health Canada's marketing authorization. "This marketing authorization will improve the visibility of sickle cell and thalassemia disorders in Canada. Access by many to this cure therapy will also reduce the stigma and discrimination experienced by many living with hemoglobinopathies in the country", advised Lanre Tunji-Ajayi, M.S.M, CEO of the GANSID.

Press release from Vertex Inc.

TORONTO, Sept. 25, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for ^PrCASGEVY^® (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). There are an estimated 2,000 patients eligible for CASGEVY in Canada, the majority of whom are living with SCD.

"Today's marketing authorization for CASGEVY as Canada's first CRISPR-based gene-editing therapy is a historic moment," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "We are excited for the potential of this one-time therapy for eligible sickle cell disease and transfusion-dependent beta thalassemia patients and are working closely with provincial, territorial and federal governments to facilitate access as rapidly as possible."

This approval is based on the positive interim results from two global and ongoing clinical trials, CLIMB-121 in SCD and CLIMB-111 in TDT. The primary endpoint of being free from severe VOCs for at least 12 consecutive months or transfusion independence for at least 12 consecutive months was met in both trials. The safety profile is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.

"CASGEVY's approval is an exciting moment for two patient communities that have long awaited an innovative therapy that brings new hope and possibilities for those in need," said Kevin Kuo, M.D., Hematologist and Clinician Investigator in the Red Blood Cell Disorders Clinic at University Health Network, and Principal Investigator for the CLIMB-131 clinical program.

Vertex has submitted CASGEVY to both Canada's Drug Agency (CDA-AMC) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Québec for Health Technology Assessments. For more information on the details of our CDA-AMC applications, including key milestones, please visit here for our SCD submission and here for our TDT submission.

The administration of CASGEVY requires specialized experience in stem cell transplantation; therefore, Vertex is working closely with provincial hospitals and health authorities to establish a network of authorized treatment centers (ATCs) to offer CASGEVY.

To read the full article, click here

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Author - vijay b