Exon skipping for 6 and 7 exons of DMD gene (preclinical)
TergetHeart: delivery of morpholinos to heart (discovery)
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.
We focus on AAV based gene delivery as AAVs look as the most promising vehicle to target human cells.
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.
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 (www.fasie.ru)
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.
WANt TO CONTACT?
Moscow, Russian Federation