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

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