Advancing Gene and Cell Therapies in South Africa

June 29, 2021 | 3 - 6:25 p.m. SAST

During this symposium, U.S. and South African speakers with experience in clinical development shared their experiences and insights on factors to consider to successfully initiate clinical trials of gene and cell therapies in South Africa. Stimulating such additional clinical research is an important step in expanding availability and access to these therapies for patients in need in the area.


3 – 3:10 p.m. SAST
Kenneth Cornetta, MD 
Indiana University 

3:10 – 4:05 p.m.
Developing Gene Therapy Clinical Trials for Monogenic Diseases 

3:10 – 3:30 p.m.
Gene Transfer for Genetic Diseases 
Johnny Mahlangu, BSc, MBBCh, MMed, FCPath 
University of the Witwatersrand and National Health Laboratory Service 

3:30 – 3:50 p.m.
BCL11A as a Molecular Target for Treatment of Sickle Cell Disease 
David Williams, MD 
Boston Children's Hospital 

3:50 – 4:05 p.m.
Q & A 

4:20 – 5:15 p.m.
Cell Therapies 

4:20 - 4:40 p.m.
Getting Cell Therapy for Leukemia and Lymphoma Into Clinical Trials in South Africa: Challenges and Opportunities 
David Brittain, MBChB, FCPath  
Albert Alberts Stem Cell Transplant Centre 

4:40 – 5 p.m.
Considerations for Developing Genetically Modified T-Cell Therapy Trials 
Marcela V. Maus, MD, PhD 
Massachusetts General Hospital 

5 – 5:15 p.m.
Q & A 

5:15 – 6:05 p.m.
Regulatory Considerations for Early Gene Therapy Trials 

5:15 – 5:35 p.m.
Health Product Regulation in South Africa 
Paul Ruff, MBBCh, MMed, FCP(SA)
Department of Medicine, University of Witwatersrand Faculty of Health Sciences 

5:35 – 5:55 p.m.
Regulatory Considerations for Gene Therapy Trials in South Africa 
Adora Ndu, PharmD, JD 
BioMarin Pharmaceutical 

5:55 – 6:10 p.m.
Q & A 

6:10 – 6:25 p.m.
Closing Remarks 
Johnny Mahlangu, BSc, MBBCh, MMed, FCPath 
University of the Witwatersrand and National Health Laboratory Service





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