ISO 15189 2022 standards for laboratory quality and competence
AGCT
1. The Power of Innovative BioTechnology
www.agct.bio
Project
Cell gene therapy for HIV cure
2. World
36 900 000
http://aidsinfo.unaids.org
The HIV crisis: solution today is a lifelong HAART
Non curable disease
poor quality of life
lower life expectancy
resistance to HAART
380 000$ lifetime costs (HAART)
indirect costs (secondary diseases)
Adapted from Lohse N, et al.
Ann Intern Med 2007;146:87–95
People living with HIV
3. Causes of death | 1987-2013
http://www.cdc.gov/hiv/library/slideSets/index.html#panel0
4. HIV infection and cancer incidence| 1991-2005
Shiels M. et al. JNCI J Natl Cancer Inst 2011;103:753-762
5. Estimated N of pts with new diagnosed lymphomas* in HIV
population – 1692 in Russia per year
Estimated N of pts with lymphomas* in HIV population, who
need to be transplanted – 790 in Russia per year
* two types only: HL+DLBCL
Auto-HSCT in lymphomas is “standard of care”
2015
6. Breakthrough technology for the HIV cure
snatched from the nature
1996: Discovery of
CCR5-delta32
2007: Berlin patient
HSC transplant from
CCR5-delta32 donor
lead to HIV cure
7. “Simple” Plan
artificial “Berlin patient” – knockout of CCR5 gene
by
genome editing in hematopoietic stem cells with
innovative high-precision and efficient technology
+
transplantation of edited autologous HSC
8. High precision disruption of CCR5 gene
with our site specific nuclease
Technology
CCR5-Uco-TALEN Targeting the CCR5 Locus
genome editing
in HSC
chemotherapy
Hematopoietic
stem cells
apheresis
infuse
Lifelong generation of
HIV-resistant
immune system
TAL Effector DNA
Binding Domain
TAL Effector DNA
Binding Domain
9. Prof. Boris Afanasyev, MD
Chairman of Clinical Advisory Board
Cell technology and HSCT
Director of large Medical University
BMT Center, St-Petersburg
Prof. Boris Feshe
CSO
Molecular biology, Genome editing
Head of the Research Cell and Gene
Therapy of the University Medical
Center Hamburg
Leading expertise on the field of
molecular biology, cell technology and HSCT
CIC 725
BMT team
10. HIVHIV Oncology
HIV in Russia
[850 000 – 1 300 000]
790 Lymphoma + HIV pts
is required to be
transplanted in Russia per
Emergency in Russia