Conference " Duchenne Muscular Dystrophy" with Marlin Biothech and Cure Duchenne

marlin biotech:next leap to treatgenetic diseases

DMD-AAV (preclinical)

SMA-AAV (preclinical)

Exon skipping for 6 and 7 exons of DMD gene (preclinical)

TergetHeart: delivery of morpholinos to heart (discovery)

Our technology

Antisense morpholino oligos

Marlin Biotech has built its own facility to produce GMP grade morpholino antisense molecules.


Marlin Biotech has successfully generated a custom model of DMD mouse with exons 8-34 deleted with CRISPR/Cas9 system.

AAV based gene delivery

We focus on AAV based gene delivery as AAVs look as the most promising vehicle to target human cells.

Exosome-based targeting

We develop a perspective method for delivery of genetic material and proteins to target tissue by exosomes. Exosomes possess higher capacity and reduced immunogenicity compared to AAV.

Our news

2018/08/21 - Start of the project "AAV-based delivery of utrophin shortened forms for DMD therapy", supported by Fund for Assistance to small innovative enterprises №359ГРНТИС5/42610 (

2018/01/10 - Marlin Biotech launched new GNAO1 project.

2017/09/01 - iCELLis Nano bioreactor installation.

2017/07/01 - Start of the development of exosome-based targeting.

2017/06/15 - Marlin Biotech and Cure Duchenne held a conference for parents and physicians.

2016/07/05 - Marlin Biotech got a resident status of Skolkovo Innovation Center.

2016/01/18 - Marlin Biotech launched a service for animal and cell models creation with CRISPR/Cas9.

2015/12/05 - Marlin Biotech cryobank of primary cell cultures from patients with Duchenne muscular dystrophy got 100th cell line!

2015/11/04 - First 5 mice with deletion of exons 8-34 were born in Marlin Biotech trasgenic mice facility.

2015/07/08 - Marlin Biotech validated internal CRISPR/Cas9 protocol for genome edited mice model creation.

2015/02/16 - Marlin Biotech submitted documents to achieve Skolkovo Innovation Center resident status.

2014/09/15 - M29 was found to be non-toxic at 12 mg/kg dose.

2014/09/09 - Marlin Biotech created a cryobank of primary cell cultures from patients with Duchenne muscular dystrophy. More than 30 cell lines are collected to the date.

2014/07/13 - First toxicology experiments with M29 have started.

2014/05/23 - We have finished screening of antisense oligonucleotides for exon 6 and 7 skipping at DMD gene. M29 drug candidate was selected for following development.


Moscow, Russian Federation