20:15 , Mar 25, 2019 |  BC Innovations  |  Distillery Therapeutics

Two promoters of FMR1 expression to treat Fragile X syndrome

DISEASE CATEGORY: Neurology INDICATION: Fragile X syndrome Cell culture and mouse studies identified two small molecule promoters of FMR1 expression that could help treat Fragile X syndrome, which is caused by FMR1 inactivation. Screening of...
13:48 , Jan 3, 2019 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Fragile X syndrome Mouse studies suggest an FMR1 peptide could help treat fragile X syndrome. In a mouse model of fragile X syndrome, a 298-mer human FMR1 peptide containing the N-terminal domain normalized the...
01:50 , Sep 14, 2018 |  BC Innovations  |  Targets & Mechanisms

Repeat offenders

Dozens of intractable DNA repeat-driven diseases could boil down to a common problem: disrupted boundaries between chromatin domains, according to a study from the University of Pennsylvania. The results present new opportunities for diseases like...
17:21 , Aug 30, 2018 |  BC Innovations  |  Tools & Techniques

Master of autism

The discovery of a master regulator of autism risk genes has yielded the first mouse model of the form of the condition that accounts for about 90% of patients, and could spark a new way...
21:45 , Jul 10, 2018 |  BC Innovations  |  Distillery Techniques

Drug delivery

TECHNOLOGY: Nanoparticles Gold-based nanoparticles could be used to deliver CRISPR-Cas9 and CRISPR-Cas12a constructs for neurological diseases. The nanoparticles are generated by conjugating gold nanoparticles to DNA oligonucleotides that bind to the CRISPR endonuclease and single-guide...
23:00 , Jun 25, 2018 |  BC Extra  |  Preclinical News

Brain-targeted CRISPR could treat autism behaviors

Researchers at University of Texas Health Science Center, University of California Berkeley and GenEdit Inc. (Berkeley, Calif.) used gold nanoparticles as a non-viral method to deliver the CRISPR gene editing complex into the brain and...
19:55 , Apr 27, 2018 |  BC Week In Review  |  Company News

Lysogene and others partner to develop gene therapy for Fragile X

Lysogene S.A. (Euronext:LYS), SATT Conectus Alsace (Illkirch, France) and Institute of Genetics and Molecular and Cellular Biology (IGBMC) (Illkirch, France) partnered to develop a gene therapy to treat fragile X. SATT Conectus said it provided...
20:32 , May 23, 2017 |  BC Innovations  |  Distillery Techniques

Disease models

TECHNOLOGY: Animal models A Drosophila larval movement-based model could be used to screen therapies for fragile X syndrome. The model involves generating Drosophila larvae at the third instar stage of development that harbor one of...
07:00 , Aug 25, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: WAS protein family member 3 (WASF3); NCK associated protein 1 (NCKAP1); cytoplasmic FMR1 interacting protein 1 (CYFIP1)

Cancer INDICATION: Breast cancer Patient sample, cell culture and mouse studies suggest that inhibiting the WASF3-NCKAP1- CYFIP1 complex could help treat metastatic breast cancer. In breast cancer patients, high tumor levels of NCKAP1 were associated...
07:00 , Jul 25, 2016 |  BioCentury  |  Finance

Leverage over genes

The decision to invest $55 million in a series A round for Fulcrum Therapeutics Inc. reflects Third Rock Ventures' belief that research tools including induced pluripotent stem cells, CRISPR- Cas9 gene editing and bioinformatics have...