agct - cell gene therapy for hiv cure
TRANSCRIPT
The Power of Innovative BioTechnology
www.agct.bio
Project
Cell gene therapy for HIV cure
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
Causes of death | 1987-2013
http://www.cdc.gov/hiv/library/slideSets/index.html#panel0
HIV infection and cancer incidence| 1991-2005
Shiels M. et al. JNCI J Natl Cancer Inst 2011;103:753-762
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
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
“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
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
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
HIV Oncology
HIV in Russia [850 000 – 1 300 000]
790 Lymphoma + HIV pts is
required to be transplanted in Russia per year
Emergency in Russia
Marina Popova
founder, CEO
www.agct.bio
The Power of Innovative BioTechnology We believe that our innovative project overturns the standard model of medical practice