This document discusses a project using gene therapy and stem cell transplantation to cure HIV. It proposes using genome editing to disrupt the CCR5 gene in hematopoietic stem cells, which would then be transplanted into patients to generate an HIV-resistant immune system. An estimated 790 people with HIV-associated lymphomas in Russia per year could benefit from this treatment. The project founders have expertise in molecular biology, genome editing, stem cell transplantation, and treating HIV/AIDS and cancer patients.

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

11. Marina Popova
founder, CEO
agct.spb@gmail.com
www.agct.bio
The Power of Innovative BioTechnology
We believe that our innovative project overturns the
standard model of medical practice